NeurologyLive® Friday 5 — March 21, 2025

Welcome to NeurologyLive^®'s Friday 5! Every week, the staff compiles 5 highlights of NeurologyLive's widespread coverage in neurology, ranging from newsworthy study findings and FDA action to expert interviews and peer-to-peer panel discussions.

1: SAT-3247 and Restoring Muscle Regeneration in Duchenne Muscular Dystrophy: Phil Lambert, PhD

The chief scientific officer at Satellos Bioscience gave clinical insight on a new novel treatment and its mechanism of action in treating Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

2: Critical Lessons From Emergence of Enzyme Replacement Therapies in Pompe Disease: Priya Kishnani, MD

The professor of pediatrics and division chief of Medical Genetics at Duke University delved into the transformative impact of enzyme replacement therapy on Pompe disease, addressing its advancements, limitations, and promising innovations shaping its future. [WATCH TIME: 2 minutes]

3: Episode 137: Advancing Neuromuscular Care and Research

In this Mind Moments^® podcast episode, former FDA commissioner Robert Califf, MD, explored the urgent need for sustained NIH funding to advance basic and translational research, including gene editing, assistive devices, and the integration of artificial intelligence. [LISTEN TIME: 9 minutes]

4: NeuroVoices: Lily Sander on Advocacy, Community, and Raising Awareness for Charcot-Marie-Tooth Disease

Patient advocate and MDA Ambassador Lily Sander shared inspiring perspective on Charcot-Marie-Tooth disease, advocacy efforts, and the importance of connection

5: Mechanism and Promise Behind Del-Zota for Duchenne Muscular Dystrophy: Michael Flanagan, PhD

The chief scientific officer at Avidity Biosciences provided clinical perspective on the function and mechanism of del-zota, an investigational antisense treatment in development for DMD amenable to exon 44 skipping. [WATCH TIME: 3 minutes]