Opinion|Videos|July 10, 2024
Targeting Pathways Downstream of Dystrophin: Vamorolone and Givinostat in Duchenne Muscular Dystrophy
Author(s)Emma Ciafaloni, MD
Emma Ciafaloni, MD, discusses therapies for Duchenne muscular dystrophy that target pathways downstream of dystrophin, focusing on vamorolone and givinostat.
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Video content above is prompted by the following questions:
- Discuss the efficacy and safety of vamorolone, a corticosteroid, which was FDA approved in 2023 for patients with Duchenne muscular dystrophy (DMD) aged 2 years and older.
- Where does givinostat, a pan-histone deacetylase inhibitor, that was FDA approved in 2024, fit in the treatment landscape of DMD?
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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.
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