This page shows what was measured, who it was measured in, and what that does not settle.
What Nusinersen does in the body
The cell then keeps the piece and builds the whole protein.
Everyone with SMA has a backup copy of the missing gene, but a single letter difference makes the cell throw away one essential piece of the instructions every time it reads them. Nusinersen is a short synthetic strand that sticks over the exact spot where the discard signal sits, hiding it. Because the molecule cannot cross from blood into the spinal cord, it has to be injected directly into the fluid around the spine.
Why people take it. Used for spinal muscular atrophy, an inherited disease that weakens muscles.
What happened in people
Thirty-seven of 73 treated babies gained a movement milestone, while none of the 37 untreated babies did.
✓ Reviewed first-read answer
Where this came from
A person wrote this and a reviewer approved it against this exact record. It carries no effect size.
A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.
No source is stored against this line.
The limit that matters most
It cannot bring back nerve cells that have already died.
Where it acts
Spinal cord motor neuron nucleus, reached through the cerebrospinal fluid
Kind of result
The kind of result is not recorded
Supervision
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.
What the registries record it as
The substance registry classes this as nucleicacid.
FDA substance registry · 5Z9SP3X666 · read 2026-08-29
Where each sentence above came from
A person wrote this explanation into the record, with the studies named in the path below.
A reviewer approved this sentence against this exact record and its sources.
The limit a reviewer approved as the one that matters most here.
The four opening statements run to 113 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.
Motor-milestone responder rate (HINE Section 2), then time to death or permanent ventilation
✓ The study showed what it set out to show
Who was studied
ENDEAR (NCT02193074)
How many people
121
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
p<0.001 for milestone response; p=0.005 for event-free survival
Repeated elsewhere
Replicated
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. Intrathecal injection of a carrier-free 2-prime-MOE phosphorothioate oligonucleotide
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
RNAWiki holds 3 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
Nusinersen
What a person takes: Intrathecal injection of a carrier-free 2-prime-MOE phosphorothioate oligonucleotide.
The measurement behind this step
Single-dose glass vials of preservative-free solution given by lumbar puncture after removal of an equivalent volume of cerebrospinal fluid. No lipid nanoparticle and no targeting ligand; the phosphorothioate backbone alone drives cellular uptake.
Getting in
Intrathecal injection into cerebrospinal fluid
The drug is injected through a needle into the fluid-filled space at the base of the spine, because it cannot travel there from the bloodstream.
╌╌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
Bolus injection into the lumbar intrathecal space. The uncharged-carrier-free phosphorothioate distributes rostrally through CSF bulk flow and reaches spinal cord grey matter and cortex, with a CNS tissue half-life of months.
Nerve cells swallow the strand without needing any packaging or carrier around 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
Gymnotic uptake by adsorptive endocytosis, driven by phosphorothioate binding to cell-surface proteins, followed by productive endosomal release into the nucleus where splicing happens.
It sits directly over the short stretch that tells the cell to throw exon 7 away, physically hiding 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 hybridisation to nucleotides 10 to 27 of SMN2 intron 7, occluding the intronic splicing silencer N1 and displacing hnRNP A1/A2 from the pre-mRNA.
With the discard signal covered, the cell assembles the instructions with the missing piece included.
╌╌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
Steric block, not cleavage: no RNase H1 is recruited. Loss of hnRNP repression restores U2AF65 and U1 snRNP recognition of the weak exon 7 splice sites, shifting the transcript pool toward full-length SMN2.
Full-length SMN protein, and motor neurons that survive
The cell makes complete, stable SMN protein, and the motor neurons that are still alive keep working.
╌╌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
Full-length SMN oligomerises and assembles the Sm core of snRNPs; the truncated SMN-delta-7 isoform is unstable and degraded. Effect is on surviving alpha motor neurons, which is why treatment timing dominates outcome.
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.
Infants, children and adults with genetically confirmed 5q SMA, increasingly including babies identified by newborn screening before any symptom appears.
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: “The safety and effectiveness of SPINRAZA in pediatric patients from newborn to 17 years have been established [see Clinical Studies ( 14.1 )].”
US prescribing information · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · read 2026-08-30
On older people, the label states: “Clinical studies of SPINRAZA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.”
US prescribing information · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no adequate data on the developmental risk associated with the use of SPINRAZA in pregnant women.”
US prescribing information · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data on the presence of nusinersen in human milk, the effects on the breastfed infant, or the effects of the drug on milk production.”
US prescribing information · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · read 2026-08-30
Where the result stopped carrying
Systemic dosing: unconjugated phosphorothioate ASOs do not cross the blood-brain barrier, forcing lifelong intrathecal administration
Non-neuronal disease features in severe SMA are not addressed by a CNS-restricted drug
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
Given by a clinician
❝ 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
Intrathecal injection of a carrier-free 2-prime-MOE phosphorothioate oligonucleotide
✎ 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. A regulator classifies this substance in a way that restricts how it is supplied.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Suppression classes recorded: S7, S11.
No source is stored against this line.
What is in the pack
Single-dose glass vials of preservative-free solution given by lumbar puncture after removal of an equivalent volume of cerebrospinal fluid. No lipid nanoparticle and no targeting ligand; the phosphorothioate backbone alone drives cellular uptake.
✎ 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
Adverse reactions in the label are dominated by the procedure rather than the molecule: lower respiratory infection, constipation, headache, back pain and post-lumbar-puncture syndrome. Thrombocytopenia, coagulation abnormality and renal toxicity are class warnings monitored on treatment.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
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
Intrathecal injection of a carrier-free 2-prime-MOE phosphorothioate oligonucleotide
✎ 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 lipid nanoparticle and no targeting ligand; the phosphorothioate backbone alone drives cellular uptake.
✎ 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
7 products list this as an active ingredient in the United States drug directory. 7 of them contain it and nothing else.
FDA National Drug Code directory · 64406-036 · read 2026-08-29
They are sold as injection, solution and powder, taken intrathecal.
FDA National Drug Code directory · 64406-036 · 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 · 64406-036 · 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 · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · read 2026-08-29
Spinraza is intrathecal at 3 DOSAGE FORMS AND STRENGTHS Injection: nusinersen is a clear and colorless solution supplied in single-dose vials in the following strengths: 12 mg/5 mL (2.4 mg/mL) 28 mg/5 mL (5.6 mg/mL) 50 mg/5 mL (10 mg/mL) Injectio…, recorded as fda label in effect 2026-03-31 in the United States.
US prescribing information · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · 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 Nusinersen 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 motor neurons already lost are restored, rather than surviving ones preserved
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 presymptomatic dosing beats symptomatic dosing in a randomised comparison; NURTURE had no control arm
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 the high-dose regimen is superior to the original dose; the DEVOTE comparator was a matched sham group from an earlier trial
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 Nusinersen 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.
ENDEAR: 51 percent of infants reached a new motor milestone, against 0 percent on sham
In plain words
In the final analysis of the sham-controlled infant trial, 37 of 73 treated infants gained a motor milestone. None of the 37 control infants did.
What was measured
Motor-milestone responders: 51 percent nusinersen versus 0 percent sham control
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Study 1 (NCT02193074) randomised 121 symptomatic infants 2:1 to nusinersen or a sham lumbar procedure. Motor-milestone response was defined on Section 2 of the Hammersmith Infant Neurologic Exam. The trial was stopped early at a pre-specified interim analysis, at which the response rate was 40 percent versus 0 percent.
Written into the record, not signed off as a reviewed claim
ENDEAR: 47 percent reduction in the risk of death or permanent ventilation
In plain words
The hard endpoint, not a score: treated infants were significantly less likely to die or end up permanently ventilated.
What was measured
Death or permanent ventilation: 47 percent risk reduction, p=0.005
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Time to death or permanent ventilation (at least 16 hours of ventilation per day for more than 21 consecutive days) was the primary endpoint of the final analysis. The label records a 47 percent risk reduction, p=0.005, with a separate significant effect on overall survival.
Written into the record, not signed off as a reviewed claim
CHERISH: 4.9-point HFMSE separation in later-onset SMA
In plain words
In older children who could already sit, treated children improved on a motor scale while sham-controlled children got worse.
What was measured
HFMSE at 15 months: +3.9 versus -1.0, p<0.0001
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Study 2 (NCT02292537) randomised 126 children 2:1. Least-squares mean change in Hammersmith Functional Motor Scale Expanded at month 15 was +3.9 (95 percent CI 3.0 to 4.9) with nusinersen against -1.0 (95 percent CI -2.5 to 0.5) with sham. 56.8 percent versus 26.3 percent achieved at least a 3-point improvement.
Written into the record, not signed off as a reviewed claim
Milestone gain is read as "reversal", which the trials did not measure
In plain words
Nusinersen keeps surviving motor neurons alive. It does not bring back motor neurons that already died, and no trial claims it does.
What was measured
That nusinersen restores normal motor development, or that it is a cure for SMA
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Both pivotal trials measured change from baseline against a concurrent control in a relentlessly progressive disease. That is a demonstration of slowed or reversed decline in the treated population, not a demonstration that lost anterior horn cells regenerate. Achieved milestones in ENDEAR were dominated by head control (22 percent) and rolling (10 percent); independent sitting was reached by 8 percent and standing by 1 percent.
Written into the record, not signed off as a reviewed claim
The field moved to treating before symptoms, on uncontrolled evidence
In plain words
Newborn screening now sends babies to treatment before any weakness appears. The study behind that shift had no control group.
What was measured
That presymptomatic superiority over symptomatic treatment has been shown in a controlled trial
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
NURTURE (NCT02386553) is an open-label, single-arm study in presymptomatic infants with two or three SMN2 copies. Its interim results, in which participants were alive without permanent ventilation and most achieved walking, drove practice and screening policy worldwide. It is a strong result against natural history, and it is not a randomised comparison.
Written into the record, not signed off as a reviewed claim
The approved dose went up, and the comparator was borrowed from an older trial
In plain words
A higher-dose regimen was added to the label. It was not compared against the original dose head to head, but against a matched sham group pulled from the 2017 infant trial.
What was measured
CHOP-INTEND at day 183: +15.1 high dose versus -11.1 matched sham
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Study 4 (NCT04089566, DEVOTE) Part B enrolled 75 infants and compared 50 patients on the high-dose regimen with a pre-specified matched sham group of 20 patients drawn from Study 1, matched on baseline disease duration and CHOP-INTEND score. The separation was large (least-squares mean difference 26.2 points, p<0.0001) and the design is external-control, not concurrent-control.
Source
SPINRAZA US prescribing information, section 14, Study 4
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
It cannot be given by injection into the bloodstream
In plain words
Phosphorothioate antisense drugs do not cross from blood into the brain and spinal cord. That is why this one needs a lumbar puncture, for life.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
Unconjugated 2-prime-MOE phosphorothioate oligonucleotides distribute to liver and kidney after subcutaneous or intravenous dosing and are excluded by the blood-brain barrier. Intrathecal administration was the engineering answer, and it carries the procedural burden and the post-lumbar-puncture adverse event profile recorded in the label. It also leaves the non-neuronal features of severe SMA untreated.
Source
SPINRAZA US prescribing information, sections 2 and 12.3
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
Where else this substance is registered
FDA substance identifier (UNII)
5Z9SP3X666
RxNorm concept
1863560
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
Suppression classes recorded: S7, S11.
✓ 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 NDA209531, approved 20161223 to BIOGEN.
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 spliceosome-blocking 18-mer injected into spinal fluid that forces the backup SMN2 gene to make full-length SMN protein; in infantile-onset SMA 51 percent of treated infants reached a new motor milestone against 0 percent of sham controls.
Recorded evidence blocks (8)
Q2
On the Nusinersen label: indicated for what?
"SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients ( 1 )": indications and usage on Nusinersen's label. DailyMed label · dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 · 2026-03-31
Q3
28 registered trials of Nusinersen — at which phases?
Registered studies posting no result
20 of 28
28 registered studies of Nusinersen: 10 na or unstated, 6 phase3, 5 phase1, 5 phase2, 1 early phase1, 1 na, 1 phase4. CLINICALTRIALS_SNAPSHOT · 2026-09-01
26 with a PubMed record
Show the evidence
na or unstated
10
phase3
6
phase1
5
phase2
5
early phase1
1
na
1
8 more recorded rows
phase4
1
completed
17
recruiting
3
active not recruiting
2
terminated
2
unknown
2
no longer available
1
withdrawn
1
recorded 2026-09-01 · last checked 2026-09-04
Q4
3 of Nusinersen's trials stopped: futility/efficacy, accrual/recruitment, other?
"After a positive interim analysis, the decision was made to terminate the study early to allow for participants to enroll into an open label study"; 3 of 28 registered studies
Show the evidence
Trial
NCT02193074
terminated; "After a positive interim analysis, the decision was made to terminate the study early to allow for participants to enroll into an open label study"
NCT02462759
terminated; "The study was terminated early to roll over participants to open label extension study NCT02594124."
NCT04050852
withdrawn; "no enrolled prospects"
recorded 2026-09-01 · last checked 2026-09-04
Q5
Nusinersen's half-life is 135 to 177 days — which schedules were studied?
135 to 177 days; Excretion The mean terminal elimination half-life is estimated to be 135 to 177 days in CSF, and 63 to 87 days in plasma.
tmaxpharmacokinetics
1.7 to 6.0 hours; Median plasma Tmax values ranged from 1.7 to 6.0 hours.
metabolismpharmacokinetics
Elimination Metabolism Nusinersen is metabolized via exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes.
recorded 2026-03-31 · last checked 2026-09-04
Q6
Which 9 trials of Nusinersen posted no result?
Posted no result
9 of 9 completed trials
Registrations
NCT01494701, NCT01780246, NCT02052791, NCT04587492, NCT04404764 and NCT04591678, and 3 more
Completion dates
oldest 2013-01-31; newest 2023-11-21
Show the evidence
Trial
NCT01494701
2013-01-31
NCT01780246
2014-02-28
NCT02052791
2017-01-31
NCT04587492
2020-09-30
NCT04404764
2021-04-23
NCT04591678
2021-06-25
3 further recorded trials
NCT03878030
2021-07-13
NCT04576494
2023-09-08
NCT04419233
2023-11-21
Q7
At the median, Nusinersen's trials enrolled 47 people — anything larger?
Median enrolment
47
Largest enrolment
1000
Registered trials counted
27
Q8
What do 425 spontaneous reports say about Nusinersen — 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.
Nusinersen appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 425 reaction mentions were counted: post lumbar puncture syndrome 70; pyrexia 64; pneumonia 64; rhinovirus infection 42. FAERS via Open Targets · CHEMBL3301572 · 2026-06-24
Show the evidence
post lumbar puncture syndrome
70
pyrexia
64
pneumonia
64
rhinovirus infection
42
respiratory tract infection
33
protein urine present
32
4 more recorded rows
scoliosis
32
respiratory failure
31
respiratory disorder
30
seizure
27
recorded 2026-06-24 · last checked 2026-09-04
Q9
Which 10 reactions does Nusinersen's label not list?
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
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