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risdiplam

Generic: RISDIPLAM·Brand: EVRYSDI, Evrysdi

risdiplam: developed by 1 company · 2 clinical trials tracked.

risdiplam evidence translation

What we can confirm

What it means

Source-backed records connect risdiplam to PTCT; that link alone does not prove the drug works.

What to watch

Watch trial status, endpoints, and the next source-backed update.
BioSniper AIPage evidence connected

Prepared research question

How could risdiplam → PTCT affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence2 indications · 1 study sponsors

Related indications

Spinal muscular atrophy (SMA)Muscular Atrophy, Spinal
Development Pipeline (1)
CompanyPhase
PTC THERAPEUTICS, INC. (PTCT)
Spinal muscular atrophy (SMA)
Approved
Clinical Trials (2)

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

NCT07047144·Phase 2·SRRK
Recruiting

Primary endpoint: Evaluate the PK of apitegromab in subjects <2 years old with SMA

InterventionalRandomizedquadruple52 targetStarted 2025-09Completion 2029-0325 sites · Belgium, France, Italy, Netherlands…Scholar Rock, Inc.

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT05808764·Phase 2·PTCT
Recruiting

Primary endpoint: Plasma Concentrations of Risdiplam

Interventionalopen10 targetStarted 2024-04Completion 2026-1113 sites · Belgium, Canada, Germany, Italy…Hoffmann-La Roche

Frequently asked questions

Who is developing risdiplam?

risdiplam is being developed by PTC THERAPEUTICS, INC..

How many clinical trials involve risdiplam?

BioSniper tracks 2 clinical trials involving risdiplam.