Opinion|Videos|July 22, 2024
Real-World Data for Risdiplam in Adult Patients with Spinal Muscular Atrophy
Panelists examine the available real-world data (RWD) on the use of risdiplam in adult patients with spinal muscular atrophy (SMA) from various studies conducted worldwide, discussing its potential efficacy and safety as a treatment option for adult SMA patients.
Advertisement
Episodes in this series
- Discuss real-world evidence for risdiplam in adult patients.
Advertisement
Related to this article
In recognition of International Ataxia Awareness Day, observed annually on September 25, NeurologyLive® reviews 6 investigational agents in clinical development across ataxia-related disorders.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 25, 2026.
One-year KYSA-6 data showed KYV-101, a CD19 CAR T-cell therapy, produced durable, drug-free responses in generalized myasthenia gravis.
The FDA has granted Scholar Rock's apitegromab fast track designation for the treatment of patients with living facioscapulohumeral muscular dystrophy as the company's phase 2 FORGE study dosing begins.
Ulefnersen met its primary end point in the phase 3 FUSION trial, providing the first placebo-controlled evidence for a genetically targeted therapy in FUS-ALS.
The CHMP recommended approval of gefurulimab (Klygefa), a weekly, self-administered C5 inhibitor, for AChR-antibody-positive generalized myasthenia gravis in the EU.
Here's some of what is coming soon to NeurologyLive® this week.
The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.
Advertisement
Advertisement
Trending on NeurologyLive - Clinical Neurology News and Neurology Expert Insights
1
What's Next in Ataxia: Pipeline Overview of Investigational Therapeutics
2
FDA Grants Fast Track Designation to Apitegromab for Facioscapulohumeral Muscular Dystrophy
3
Azetukalner NDA Submitted, EMA Gives Approval Recommendation to Ocrelizumab, Phase 3 FUSION Trial Meets End Points
4
Ulefnersen Meets Primary End Point in Phase 3 FUSION Trial for FUS-ALS
5