Last reviewed · How we verify

NCT04256265

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

APPROVED FOR MARKETING Last updated 5 October 2020
What this trial tests

trial testing Risdiplam in Muscular Atrophy, Spinal. Approved for marketing.

Quick facts

Lead sponsorGenentech, Inc.
StatusAPPROVED FOR MARKETING
Study typeEXPANDED_ACCESS
Sites29 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Genentech, Inc. — full company profile →

Who can join

2 Months and older, any sex, with Muscular Atrophy, Spinal. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

  1. New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges.
    Messina S, Sframeli M. · · 2020 · cited 86× · PMID 32668756 · DOI 10.3390/jcm9072222
  2. An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.
    Kwon JM, Arya K, Kuntz N, Phan HC, et al · · 2022 · cited 31× · PMID 35567422 · DOI 10.1002/acn3.51560
  3. Risdiplam in non-sitter patients aged 16 years and older with 5q spinal muscular atrophy.
    Ñungo Garzón NC, Pitarch Castellano I, Sevilla T, Vázquez-Costa JF. · · 2023 · cited 27× · PMID 36815750 · DOI 10.1002/mus.27804
  4. Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment.
    Menduti G, Rasà DM, Stanga S, Boido M. · · 2020 · cited 27× · PMID 33281605 · DOI 10.3389/fphar.2020.592234
  5. Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape.
    Ponomarev AS, Chulpanova DS, Yanygina LM, Solovyeva VV, et al · · 2023 · cited 22× · PMID 37762045 · DOI 10.3390/ijms241813743
  6. Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical-Experimental, and Real-World Evidence.
    Crisafulli S, Boccanegra B, Vitturi G, Trifirò G, et al · · 2023 · cited 11× · PMID 37891814 · DOI 10.3390/brainsci13101446
  7. Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.
    Svoboda MD, Kuntz N, Leon-Astudillo C, Byrne BJ, et al · · 2025 · cited 3× · PMID 40624473 · DOI 10.1186/s12883-025-04276-4

Verify or expand the search:

Other trials of Risdiplam

Trials testing the same drug.

Other recruiting trials for Muscular Atrophy, Spinal

Currently open trials in the same condition.

Other Genentech, Inc. trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04256265.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing