This page shows what was measured, who it was measured in, and what that does not settle.
What Golodirsen does in the body
Duchenne Muscular Dystrophy (exon 53 skipping)
Golodirsen covers exon 53 of the dystrophin instructions so the cell leaves it out. For boys whose deletion sits next to exon 53, that realigns the reading frame and lets a shortened dystrophin be built. The measured amount of protein produced is around one percent of a healthy person, and the trial designed to show whether that changes how boys move did not find a difference.
What happened in people
Exon 53 skipping increases in treated muscle, confirmed by blinded RT-PCR
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The recorded finding that sits closest to something a person would notice. Written into the record, not signed off, and it carries no population or interval.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
The limit that matters most
That around 1 percent of normal dystrophin translates into slower functional decline
Where it acts
Skeletal muscle fibre nucleus
Kind of result
A step measured inside a person
Supervision
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
What the registries record it as
The substance registry classes this as nucleicacid.
FDA substance registry · 033072U4MZ · read 2026-08-29
Where each sentence above came from
No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.
Shown as the opening line on this page.
A limit recorded against this substance. Not signed off as a reviewed claim.
The four opening statements run to 85 words.
Words this page uses
Four words worth knowing first
Chosen from what this page shows, with each one explained before the word it depends on.
Protein
A protein is a folded chain your body builds to do a specific job.
A picture of it, and where the picture fails
A protein is like a tool bent into one shape for one task.
Where that stops being true. A tool keeps its shape. A protein can change shape and stop working.
What people get wrong. Protein in food and a protein in the body are related but not the same thing.
A polymer of amino acids folded into a defined structure that determines its function.
Gene
A gene is a stretch of instructions for building one protein.
A picture of it, and where the picture fails
A gene is like one page of a building plan.
Where that stops being true. A page is read the same way every time. A gene can be read more or less often.
What people get wrong. Having a gene is often read as having a trait. Whether it is used matters as much.
A segment of DNA that encodes a functional product, usually a protein.
Messenger RNA
Messenger RNA is a working copy of one gene, carried to where proteins are built.
A picture of it, and where the picture fails
It is like a photocopy of one plan page, taken to the workshop.
Where that stops being true. A photocopy lasts. This copy is destroyed soon after use, on purpose.
What people get wrong. It is often thought to change the gene. It is a copy, and it does not alter the original.
A single-stranded transcript of a gene that ribosomes translate into a protein.
Small interfering RNA
A small interfering RNA is a short piece that makes a cell destroy one working copy.
A picture of it, and where the picture fails
It is like a note telling the workshop to shred one plan page.
Where that stops being true. A note is read once. This keeps working for months after one injection.
What people get wrong. It is often described as gene editing. It leaves the gene untouched.
A short double-stranded RNA that directs the RNA-induced silencing complex to cleave a complementary transcript.
What happened in people◇Read from sources, not yet reviewed
What happened in people
Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.
Change from baseline in muscle dystrophin protein by western blot at week 48 of Part 2
✓ The study showed what it set out to show
Who was studied
Study 4053-101 (NCT02310906)
How many people
25
Study design
Phase 1/2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
p<0.001
Repeated elsewhere
Unreplicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous infusion of an uncharged phosphorodiamidate morpholino oligomer
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
FDA summary review, NDA 211970 (golodirsen), including the August 2019 complete response letter (https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/2119… · a recorded source, not a stored snapshot
Change from baseline in 4-step ascend velocity at week 96 versus placebo
✗ The study did not show it
Who was studied
ESSENCE (NCT02500381)
How many people
212
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
p=0.309 (LSM difference 0.06 steps/s, 95 percent CI -0.05 to 0.16)
Repeated elsewhere
Failed to Replicate
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. None new reported through week 144; the trial pooled golodirsen and casimersen arms for the primary comparison
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous infusion of an uncharged phosphorodiamidate morpholino oligomer
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
FDA summary review, NDA 211970 (golodirsen), including the August 2019 complete response letter (https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/2119… · a recorded source, not a stored snapshot
What we know
RNAWiki holds 2 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What it changes in the body◇Read from sources, not yet reviewed
The path through the body
From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.
Start
Golodirsen
What a person takes: Intravenous infusion of an uncharged phosphorodiamidate morpholino oligomer.
The measurement behind this step
Single-dose vials diluted into saline, infused weekly over 35 to 60 minutes. No carrier and no targeting ligand; long-term venous access is usual.
Getting in
Weekly intravenous infusion
Given into a vein once a week, indefinitely.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Uncharged 25-mer PMO in isotonic phosphate-buffered saline. Distribution is broad but muscle uptake is a small fraction of dose; most drug is cleared renally within hours.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
Reaching the cell
Uptake into muscle fibre nuclei
A small proportion of the drug crosses into muscle cells and reaches the nucleus, where splicing happens.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Neutral backbone gives nuclease resistance at the cost of the protein-binding uptake route available to phosphorothioates. Dystrophic membrane fragility is thought to contribute to what uptake there is.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
What it acts on
Hybridising across exon 53
It pairs with exon 53 in the dystrophin instructions and masks it.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Watson-Crick pairing to a 25-nucleotide site within DMD exon 53, blocking exonic splicing enhancer recognition and preventing exon definition.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
The change it makes
Exon 53 is spliced out and the frame is restored
The cell joins the exons on either side, which realigns the reading frame for the right deletions.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
Exclusion of exon 53 produces an in-frame junction in patients with deletions such as 52, 45-52 or 50-52, permitting translation through the cysteine-rich and C-terminal domains.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
What that does for a person
Truncated dystrophin at the sarcolemma, around one percent of normal
Shortened dystrophin does appear at the right place in the muscle membrane, in about one hundredth of the normal quantity.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Immunohistochemistry confirms sarcolemmal localisation; western blot quantifies the mean at 1.02 percent of healthy muscle. Whether this reconstitutes enough dystrophin-glycoprotein complex to reduce contraction-induced injury remains unmeasured.
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
No suggested links are held for this record, so nothing is hidden from this path.
What we know
The record describes 5 steps from taking it to an effect in a person.
How we know it
Each step names the study behind it in the technical detail beside it.
What this does not prove
A change inside the body is a reason to look. It is not a result in a person.
Why it matters
A reader can see exactly where the chain stops being measured in people.
What we still do not know
No reviewed claim binds any of these steps to a named population.
What is missing or unclear◇Read from sources, not yet reviewed
Were people like you studied?
RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.
Who was studied
People similar to this profile were included.
Boys and young men with an exon-53-amenable DMD deletion, by weekly intravenous infusion, on top of corticosteroids.
Who is missing from the studies
Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What the label states about particular groups
On pediatric, the label states: “VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, including pediatric patients [see Clinical Studies ( 14 )].”
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-30
On older people, the label states: “DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with VYONDYS 53.”
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no human or animal data available to assess the use of VYONDYS 53 during pregnancy.”
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no human or animal data to assess the effect of VYONDYS 53 on milk production, the presence of golodirsen in milk, or the effects of VYONDYS 53 on the breastfed infant.”
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-30
Where the result stopped carrying
The FDA issued a complete response letter in August 2019 over renal toxicity in animals and vascular-access infection risk
ESSENCE, the confirmatory randomised trial, missed its primary functional endpoint in 2026
This is a scope explorer, not a diagnosis engine.
It shows who was studied so you can see whether people similar to you were included.
RNAWiki cannot tell whether a study fits you. It can show who was in it.
What it would be like to take◇Read from sources, not yet reviewed
Why it might seem to do nothing
A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.
It was studied in different people
The people in the studies were not much like the reader.
On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
The evidence may simply be wrong
Small early studies often do not hold up.
On this record: RNAWiki has not yet published a reviewed conclusion for this use.
Other reasons RNAWiki checked and found nothing for (11)
It was studied for a different goal
The studies measured something else entirely. Nothing in this record points to this reason.
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.
None of these is a reason to take more. Taking more is not a step this page ever suggests.
What it would be like to take◇Read from sources, not yet reviewed
What taking it involves
The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.
How it is supplied
Not established
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Read from the recorded approval status.
No source is stored against this line.
Form and route
Intravenous infusion of an uncharged phosphorodiamidate morpholino oligomer
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form and route recorded on this substance.
No source is stored against this line.
Who oversees it
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
The identity record classes it as ASO (Antisense Oligonucleotide).
No source is stored against this line.
What is in the pack
Single-dose vials diluted into saline, infused weekly over 35 to 60 minutes. No carrier and no targeting ligand; long-term venous access is usual.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on how this is sold and what that means for what is in the pack.
No source is stored against this line.
Where it is registered
Regulatory records are listed in the technical disclosure at the foot of this page.
# Counted from records
Where this came from
A count of rows RNAWiki holds. It describes our records, not your body.
Register entries are stored per jurisdiction and shown with their dates.
No source is stored against this line.
Why people stop
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No record of why people stopped taking it is stored for this substance.
No source is stored against this line.
What it would be like to take◇Read from sources, not yet reviewed
What can go wrong
Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.
·Worth asking a clinician aboutWritten into the record from the studies named on this page
Labelled warning for renal toxicity based on nonclinical findings, with monitoring of serum cystatin C, urine dipstick and urine protein-to-creatinine ratio. Hypersensitivity reactions labelled. Common adverse reactions include headache, pyrexia, cough, abdominal pain and nausea.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
Where this came from
VYONDYS 53 (golodirsen) US prescribing information, DailyMed (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5) · a recorded source, not a stored snapshot
Muntoni et al., Efficacy and Safety of Golodirsen and Casimersen versus Placebo (ESSENCE): phase 3 topline results, MDA 2026 (https://www.mdaconference.org/a… · a recorded source, not a stored snapshot
Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.
Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What is missing or unclear◇Read from sources, not yet reviewed
Does the exact form matter?
Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.
The form that was studied
Intravenous infusion of an uncharged phosphorodiamidate morpholino oligomer
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
No carrier and no targeting ligand; long-term venous access is usual.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on which forms are sold and how they compare.
No source is stored against this line.
What is recorded as being sold
4 products list this as an active ingredient in the United States drug directory. 4 of them contain it and nothing else.
FDA National Drug Code directory · 60923-465 · read 2026-08-29
They are sold as injection and powder, taken intravenous.
FDA National Drug Code directory · 60923-465 · read 2026-08-29
The regulator's established pharmacologic class for it is antisense oligonucleotide [epc], antisense [cs] and increased protein synthesis [pe].
FDA National Drug Code directory · 60923-465 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-29
Vyondys 53 is intravenous at 3 DOSAGE FORMS AND STRENGTHS VYONDYS 53 is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles, and available as: Injection: 100 mg/2 mL (50 m…, recorded as fda label in effect 2025-03-13 in the United States.
US prescribing information · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · read 2026-08-30
Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.
What it would be like to take◇Read from sources, not yet reviewed
What you could measure
This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.
Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.
Questions worth asking
Which of the trials of Golodirsen studied people like me?
What was measured, and for how long?
Was the result a laboratory value or a health outcome?
What would we watch for, and when would we stop?
Tracking can show whether something changed for you. It cannot show what caused it.
RNAWiki records evidence. It does not say whether this substance is right for you.
What is missing or unclear◇Read from sources, not yet reviewed
Claims that go past the evidence
Things said about this substance that sound reasonable, and the exact step that is missing between the evidence and the claim.
✗Goes past the evidence
That around 1 percent of normal dystrophin translates into slower functional decline
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That viltolarsen is more potent because it reports a higher percentage; the assays differ
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
What is missing or unclear◇Read from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
How much did people take in the studies?
The sources RNAWiki checked hold nothing for this field.
Why it matters. A result belongs to an amount. Without the amount the result floats free.
What would answer it
A stored source that records it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing populations
Which groups were under-represented in the studies has not been recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing long-term data
No completed tested study window is recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
No reviewed conclusion
RNAWiki has not yet published a reviewed conclusion for this use.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing interaction studies
No interaction was found in the registers checked. Not finding one is not the same as showing there is none.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Formulation uncertainty
Several salts, forms or products of Golodirsen are recorded. Results from one form may not transfer to another.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Mechanism not reviewed
No reviewed mechanism story exists for this substance.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
What happened in people◇Read from sources, not yet reviewed
Check any of this yourself
Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.
Every line above can be traced to the study named beside it. Follow the link and read it.
Dystrophin rose from 0.10 to 1.02 percent of normal at week 48
In plain words
Twenty-five boys were biopsied before treatment and again after 48 weeks. Their average dystrophin went from a tenth of a percent to about one percent of normal.
What was measured
Mean dystrophin 0.10 percent to 1.02 percent of normal, p<0.001, n=25
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Study 1 (NCT02310906) Part 2 biopsied all 25 treated patients at baseline and week 48. Mean dystrophin was 0.10 percent (SD 0.07) at baseline and 1.02 percent (SD 1.03) at week 48, mean change 0.92 percent (SD 1.01), p<0.001, median change 0.88 percent. Individual week-48 values in the label range from 0.09 to 4.30 percent.
Written into the record, not signed off as a reviewed claim
Exon 53 skipping increased about 16-fold over baseline
In plain words
The mechanism was confirmed directly: treated muscle showed far more of the skipped transcript, and the extra protein appeared at the right place on the muscle membrane.
What was measured
Approximately 16-fold increase over baseline dystrophin at week 48
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Blinded RT-PCR showed a significant increase in exon 53 skipping, associated with an approximately 16-fold increase over baseline in dystrophin protein at week 48. Immunohistochemistry showed increased dystrophin-positive fibres (p<0.001) and correlated with western blot (Spearman r=0.663, p<0.001).
Written into the record, not signed off as a reviewed claim
ESSENCE, the confirmatory trial, missed its primary endpoint
In plain words
The randomised trial that was supposed to prove the drug helps boys move reported in 2026. There was no significant difference from placebo.
What was measured
4-step ascend velocity: LSM difference 0.06 steps/s, 95 percent CI -0.05 to 0.16, p=0.309
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
ESSENCE (NCT02500381) randomised 212 ambulatory patients to golodirsen or casimersen versus placebo over 96 weeks. The primary endpoint, change in 4-step ascend velocity, gave a least-squares mean difference of 0.06 steps per second (95 percent CI -0.05 to 0.16, p=0.309). Secondary functional measures including the 6-minute walk test, 10-metre walk/run and North Star Ambulatory Assessment favoured treatment numerically without reaching significance. Dystrophin expression again increased significantly.
Source
Muntoni et al., ESSENCE phase 3 topline results, MDA Clinical and Scientific Conference 2026
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
contested
Review state
Written into the record, not signed off as a reviewed claim
One percent of normal dystrophin is treated as a therapeutic level
In plain words
Nobody has established how much dystrophin a muscle needs before it stops being damaged. The approval assumed that more is better without knowing where the line is.
What was measured
That raising dystrophin to about 1 percent of normal slows functional decline in Duchenne
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Accelerated approval was granted on dystrophin as a surrogate reasonably likely to predict clinical benefit. The distribution matters as much as the mean: in the label table, 7 of 25 patients ended below 0.25 percent of normal at week 48. With the confirmatory functional endpoint now missed, the surrogate-to-outcome link is unsupported by randomised evidence.
Written into the record, not signed off as a reviewed claim
Rejected in August 2019 on safety, approved four months later
In plain words
The FDA first refused the application over kidney toxicity seen in animals and over infections from long-term infusion lines. After a formal dispute, the same agency approved it in December 2019.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
A complete response letter issued on 19 August 2019 cited risk of infections related to vascular access ports and renal toxicity observed in mice, rats and monkeys. The sponsor filed a formal dispute resolution request. Approval followed on 12 December 2019, with renal toxicity retained as a labelled warning and no new clinical safety data resolving the animal finding.
Source
FDA summary review, NDA 211970; Sarepta approval announcement December 2019
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
Cross-drug dystrophin comparisons with viltolarsen are not valid
In plain words
Viltolarsen reports 5.9 percent and golodirsen reports 1.02 percent for the same exon. That gap is mostly assay, not biology.
What was measured
That viltolarsen produces roughly six times more dystrophin than golodirsen
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Golodirsen was quantified by Sarepta western blot against a healthy-muscle standard; viltolarsen was quantified by western blot normalised to myosin heavy chain, with mass spectrometry normalised to filamin C as a secondary measure. Different antibodies, different normalisation proteins and different standard curves. No head-to-head study has ever measured the two drugs on one assay.
Source
VYONDYS 53 and VILTEPSO US prescribing information, section 14 methods statements
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
Where else this substance is registered
FDA substance identifier (UNII)
033072U4MZ
RxNorm concept
2267211
Checks this page had to pass
✓ Passed
Identity resolved
no open identity hold
✓ Passed
No unresolved merge across substance families
no quarantine open
✓ Passed
Every public sentence names a source
The opening statement carries the origin: Written into the record, not signed off.
✗ Not passed
Trial roles classified for highlighted evidence
No registered study is classified as testing this substance.
✓ Passed
No internal keys in reader text
enforced by the copy-contract test over the rendered page
✓ Passed
Safety mode resolved
The identity record classes it as ASO (Antisense Oligonucleotide).
✓ Passed
Canonical metadata present
slug and display name present
What is missing or unclear◇Read from sources, not yet reviewed
How this medicine reached us
Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.
What the approval register records
1 approved application covers products containing this substance. The earliest was NDA211970, approved 20191212 to SAREPTA THERAPS INC.
This order is fixed in code and does not count clicks or time on the page.
What is not here
7 questions this page could not answer
These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.
What was measured, goal by goal — found nothing in the sources checked.
How close this is to real life — found nothing in the sources checked.
Felt, measured, or meaningful — found nothing in the sources checked.
How long anything takes — found nothing in the sources checked.
What it may clash with — found nothing in the sources checked.
Other ways to the same goal — found nothing in the sources checked.
What changed on this page — found nothing in the sources checked.
The record as stored
The full record, for auditing
Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.
The older medicine-wide conclusion held in this record
Written for a clinical reader, and about the medicine as a whole rather than about one indication, population and set of trials. RNAWiki does not treat it as a programme conclusion and no reviewer has signed it off in that form. It is here word for word because it is what the record holds.
A 25-mer morpholino for exon 53 skipping that raised muscle dystrophin from 0.10 to 1.02 percent of normal in 25 boys, and whose confirmatory trial reported in 2026 with no significant functional separation from placebo.
Recorded evidence blocks (8)
Q1
On the Golodirsen label: indicated for what?
"VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients…": indications and usage on Golodirsen's label. DailyMed label · 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5 · 2025-03-13
Q2
5 registered trials of Golodirsen — at which phases?
"The trial was stopped to reduce the clinical trial burden on participants while ensuring continued treatment via a post-trial access program with commercially available casimersen and golodirsen. Study was not terminated due to safety concerns."; 1 of 5 registered studies
Show the evidence
TrialNCT03532542
terminated; "The trial was stopped to reduce the clinical trial burden on participants while ensuring continued treatment via a post-trial access program with commercially available casimersen and golodirsen. Study was not terminated due to safety…"
recorded 2026-09-01 · last checked 2026-09-04
Q4
Golodirsen's half-life is 3.4 hours — which schedules were studied?
3.4 hours; The elimination half-life (t 1/2 ) was 3.4 hours.
metabolismpharmacokinetics
Metabolism Golodirsen is metabolically stable.
recorded 2025-03-13 · last checked 2026-09-04
Q5
At the median, Golodirsen's trials enrolled 171 people — anything larger?
Median enrolment
171
Largest enrolment
300
Registered trials counted
5
Q6
What do 57 spontaneous reports say about Golodirsen — and not say?
These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.
Golodirsen appears in spontaneous reports to regulators. Across the 4 most-reported reaction terms, 57 reaction mentions were counted: product dose omission issue 35; poor venous access 17; exposure to sars-cov-2 3; catheter site discolouration 2. FAERS via Open Targets · CHEMBL4297762 · 2026-06-24
Show the evidence
product dose omission issue
35
poor venous access
17
exposure to sars-cov-2
3
catheter site discolouration
2
recorded 2026-06-24 · last checked 2026-09-04
Q7
Which 4 reactions does Golodirsen's label not list?
Drug Interaction Studies Golodirsen did not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5 in vitro .
pharmacokinetics
Golodirsen was a weak inducer of CYP1A2 and did not induce CYP2B6 or CYP3A4.
pharmacokinetics
Golodirsen was not metabolized by human hepatic microsomes and was not a substrate or strong inhibitor of any of the key human drug transporters tested (OAT1, OAT3, OCT2, OATP1B1, MATE1, P-gp, BCRP, and MRP2, OATP1B3 and MATE2-K).
clinical_pharmacology
Drug Interaction Studies Golodirsen did not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5 in vitro .
clinical_pharmacology
Golodirsen was a weak inducer of CYP1A2 and did not induce CYP2B6 or CYP3A4.
clinical_pharmacology
Golodirsen was not metabolized by human hepatic microsomes and was not a substrate or strong inhibitor of any of the key human drug transporters tested (OAT1, OAT3, OCT2, OATP1B1, MATE1, P-gp, BCRP, and MRP2, OATP1B3 and MATE2-K).
ChEMBL 37 — CC BY-SA 3.0 Unported · ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Open Targets 26.06 — CC0 · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
✗ required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
✓ public claims reviewed: 0 reviewed claim(s); drafts are never rendered
✓ source coverage passed: 5 source rows
✓ no critical contamination: no quarantine open
✓ canonical metadata passed: slug and display name present
✓ no raw internal fields: enforced by the copy-contract test over the rendered page
This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.