NeurologyLive® Friday 5 — March 24, 2023

Article

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 24, 2023.

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: FDA AdComm on Tofersen Supports NfL as Surrogate End Point of Clinical Benefit, But Is It Enough for Approval?

In a meeting of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee to review tofersen (Biogen), an investigational antisense oligonucleotide currently under review for SOD1 mutated-mediated amyotrophic lateral sclerosis (ALS), the committee voted unanimously that the reduction in plasma neurofilment light chain (NfL) concentration in tofersen-treated patients is reasonably likely to predict clinical benefit of tofersen; however, the committee did not vote in support of the clinical data providing substantial evidence of effectiveness relevant to full approval of tofersen.

FDA AdComm on Tofersen Supports NfL as Surrogate End Point of Clinical Benefit, But Is It Enough for Approval?

2: Peer Exchange: Overview of Neuromyelitis Optica Spectrum Disorder (NMOSD)

In episode 1 of this Peer Exchange series on "Updates in the Management of Neuromyelitis Optica Spectrum Disorder Care (NMOSD)", experts in neurology provide an overview of NMOSD and discuss recent approvals and updates in the treatment and management of this condition.

Overview of Neuromyelitis Optica Spectrum Disorder (NMOSD)

3: NeuroVoices: Emma Ciafaloni, MD, on Mounting Progress in the Neuromuscular Field, Duchenne Muscular Dystrophy

In the latest edition of our in-depth Q&A series, the professor of neurology and pediatrics at University of Rochester Medical Center shared her thoughts on the 2023 MDA Clinical and Scientific Conference, and the progress made in the treatment of neuromuscular disorders.

NeuroVoices: Emma Ciafaloni, MD, on Mounting Progress in the Neuromuscular Field, Duchenne Muscular Dystrophy

4: Episode 85: Impact of Narcolepsy on Social Relationship Health in Adolescents

In episode 83 of the Mind Moments Podcast features an exclusive interview with Eric Zhou, PhD, assistant professor of pediatrics at Harvard Medical School. He spoke on a recently conducted study assessing the link between narcolepsy in adolescents and social relationship health.

Episode 85: Impact of Narcolepsy on Social Relationship Health in Adolescents

5: Turning Gene Therapy Into Reality: Peter Marks, MD, PhD

The director of the Center for Biologics Evaluation Research at the FDA provided an overview of his Keynote Address he gave at the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, and the reasons for why he chose this topic. He spoke on the steps needed to continue the advancement of gene therapies, and why speaking to the MDA community can have major impacts. [WATCH TIME: 3 minutes]

Turning Gene Therapy Into Reality: Peter Marks, MD, PhD
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