Browse medicines
Some records contain detailed evidence; others currently contain only basic identity and regulatory information. Reviewed conclusions appear only when they are available for a specific use and group of people.
Showing 1–14 of 14 records
Inotersen (Tegsedi)
Gene-silencing medicineApproved in the United States (FDA)Detailed recordUse listed on the medicine record
Hereditary ATTR Amyloidosis with Nerve Damage
Nusinersen (Spinraza)
Gene-silencing medicineApproved in the United States (FDA)Detailed recordUse listed on the medicine record
Spinal Muscular Atrophy
Eteplirsen (Exondys 51)
Gene-silencing medicineFDA accelerated approval (based on an earlier result expected to predict benefit; follow-up evidence may be required)Detailed recordUse listed on the medicine record
Duchenne Muscular Dystrophy (exon 51 skipping)
Golodirsen (Vyondys 53)
Gene-silencing medicineFDA accelerated approval (based on an earlier result expected to predict benefit; follow-up evidence may be required)Detailed recordUse listed on the medicine record
Duchenne Muscular Dystrophy (exon 53 skipping)
Eplontersen (Wainua)
Gene-silencing medicineApproved in the United States (FDA)Detailed recordUse listed on the medicine record
Hereditary ATTR Amyloidosis with Nerve Damage
Tofersen (Qalsody)
Gene-silencing medicineFDA accelerated approval (based on an earlier result expected to predict benefit; follow-up evidence may be required)Detailed recordUse listed on the medicine record
SOD1 Amyotrophic Lateral Sclerosis (ALS)
Casimersen (Amondys 45)
Gene-silencing medicineFDA accelerated approval (based on an earlier result expected to predict benefit; follow-up evidence may be required)Detailed recordUse listed on the medicine record
Duchenne Muscular Dystrophy (exon 45 skipping)
Volanesorsen (Waylivra)
Gene-silencing medicineApproved in the European Union (EMA)Detailed recordUse listed on the medicine record
Familial Chylomicronaemia Syndrome (very high triglycerides)
Viltolarsen (Viltepso)
Gene-silencing medicineFDA accelerated approval (based on an earlier result expected to predict benefit; follow-up evidence may be required)Detailed recordUse listed on the medicine record
Duchenne Muscular Dystrophy (exon 53 skipping)
Mipomersen (Kynamro)
Gene-silencing medicineApproved in the United States (FDA)Detailed recordUse listed on the medicine record
Homozygous Familial Hypercholesterolaemia (withdrawn)
Aprinocarsen (Dr Dale Wellness Nu Fem 1 2 3 All in One Topical Homeopathic)
Gene-silencing medicineDietary supplement; not FDA-approved as a medicineExpanded recordUse listed on the medicine record
Topical gel, applied to skin
Fomivirsen (Vitravene Preservative Free)
Gene-silencing medicineApproved in the United States (FDA)Expanded recordDonidalorsen (Dawnzera (Autoinjector) / Dawnzera)
Gene-silencing medicineApproved in the United States (FDA)Expanded recordUse listed on the medicine record
Prophylaxis to prevent attacks of hereditary angioedema
Olezarsen (Tryngolza (Autoinjector) / Tryngolza)
Gene-silencing medicineApproved in the United States (FDA)Expanded recordUse listed on the medicine record
Triglycerides