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Protein target · ChEMBL and the Guide to Pharmacology

Medicines acting on SMN2

Survival of motor neuron 2, centromeric. Every medicine in our data that ChEMBL or the Guide to Pharmacology records as acting on this protein, with the source for each and its current clinical trials on this site.

Sources: ChEMBL (CC BY-SA 3.0) via the Open Targets Platform, release 26.09; IUPHAR/BPS Guide to PHARMACOLOGY 2026.3 (CC BY-SA 4.0). ENSG00000205571
3Medicines acting on SMN2 (3 with a page here)
0 of 3Listed by both ChEMBL and the Guide to Pharmacology
10Current clinical trials of these medicines in our registers
Positive modulatorMost common way they act (2 of 3)
01

Medicines acting on SMN2

most-trialled first
MedicineHow it actsSourceCurrent trials here
Risdiplam
CHEMBL4297528
SMN2 pre-mRNA positive modulator Positive modulator
Small molecule
 ChEMBL only 6
Nusinersen
CHEMBL3301572
SMN2 pre-mRNA positive modulator Positive modulator
Oligonucleotide
 ChEMBL only 4
Onasemnogene Abeparvovec
CHEMBL4297240
Survival motor neuron protein exogenous gene Exogenous gene
Gene
 ChEMBL only —

“Both” means ChEMBL and the Guide to Pharmacology each record this medicine acting on this protein; “only” means one source records it and the other does not (often because it lists fewer secondary targets). The Guide to Pharmacology adds a medicine here only when this protein is its primary target. Medicines without a link have no page on this site yet.

02

Diseases these medicines are tested for

by number of SMN2 medicines
DiseaseMedicines testedFurthest trial phase
Proximal spinal muscular atrophy 3 Phase 3
Spinal muscular atrophy, type 1 2 Phase 1/2
Metabolic dysfunction-associated steatohepatitis 1 Phase 1
Non-alcoholic fatty liver 1 Phase 1

From ChEMBL's drug-disease records, which draw on trial registries. A trial phase is not an approval; approved uses are on each medicine's page from the regulators' own records.

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How this page is built

Which medicines act on this protein comes from two independent, expert-curated sources: ChEMBL's mechanism-of-action records (via the Open Targets Platform) and the IUPHAR/BPS Guide to PHARMACOLOGY. Each row says which of them lists it. Data from them is shared here under their share-alike licences (CC BY-SA 3.0 and 4.0).

A job re-reads both sources every 30 days. Medicines are matched to our pages by exact name or a ChEMBL-curated synonym, and to the Guide to Pharmacology by ChEMBL ID or exact name, never by a close spelling. No AI drafting. See our methodology. The Open Targets data on this page was last retrieved on 30 Sep 2026. How every register is built: methodology · fixes we have made: corrections log.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.