Special Episode: SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy

Natalie Goedeker, CPNP

A special episode of the NeurologyLive^® Mind Moments^® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service.

The Mind Moments^® podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis (MS), Parkinson disease, dementia, sleep disorders, and more.

This episode, "SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy," features an exclusive interview with Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis. SRP-9001 (Elevidys; Sarepta), also known as delandistrogene moxeparvovec, was approved for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene aged 4 to 5 years, based on data from the 3 clinical trials.¹

Click here to read more of NeurologyLive^®'s coverage of the SRP-9001 approval.

EPISODE BREAKDOWN

• 0:30 – SRP-9001 (Elevidys; Sarepta) is approved for DMD
• 1:35 – Natalie Goedeker, CPNP, on the approval
• 3:05 – Efficacy of SRP-9001
• 4:00 – Goedeker on pieces of efficacy data released
• 4:45 – Currently available therapies for DMD
• 5:35 – Goedeker on role of SRP-9001 in treatment landscape
• 6:55 – Safety data of SRP-9001
• 8:00 – Goedeker on safety considerations of which prescribing clinicians should be aware

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REFERENCE
1. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed June 22, 2023. https://www.businesswire.com/news/home/20230622454844/en/