Neuromuscular

Basil Darras, MD discusses the FIREFISH trial of risdiplam in infants with type 1 symptomatic SMA and comments on the difficulty of comparing agents when trials have had different enrollment criteria.

Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.

The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital discussed how the PHOENIX study expands upon the previous study, CENTAUR.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending May 28, 2021.

Six months after being discharged from the hospital for COVID-19, patients scored worse than average on 1 or more NeuroQoL scale components, which measured anxiety, sleep, fatigue, and depression.

The long-term follow up of the phase 1 START trial found that previously achieved motor milestones were preserved over a 5-year period.

Matthew B. Harms, MD, associate professor of neurology at Columbia and medical consultant for the Muscular Dystrophy Association, shares his thoughts on the current state of ALS care.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending May 21, 2021.

"Mind Moments," a podcast from NeurologyLive, brings you an exclusive interview with Matthew B. Harms, MD.

The trial design was presented at the 2021 ENCALS meeting and is set to run for 48 weeks at more than 50 clinical centers.

The investigational drug, previously granted fast-track designation, did not meet primary or secondary end points in the ORARIALAS-01 trial.

Copy number results for the kit were highly concordant with reference results from other methods.

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The 2 organizations will use whole-genome sequencing and clinical characterization to investigate the genetic causes of CP.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending May 14, 2021.

Data presented at the ASGCT 2021 meeting showed increased microdystrophin expression with SGT-001.

The partnership combines Capsigen’s vector engineering with Biogen’s drug pipeline, seeking to develop gene therapies for neuromuscular and central nervous system disorders.

The associate professor of neurology at Columbia University and medical consultant and care center director at the Muscular Dystrophy Association spoke to the importance of staying up on the latest developments in ALS and keeping patients in the loop on that information.

Here's what is coming soon to NeurologyLive.

The associate professor of neurology at Columbia University and medical consultant and care center director at the Muscular Dystrophy Association discussed the use of larger datasets to improve prognostication and clinical trials in ALS.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending May 7, 2021.

The associate professor of neurology at Columbia University and medical consultant and care center director, Muscular Dystrophy Association, discusses the impact that the Ice Bucket Challenge and ALS Awareness Month have had on the ALS treatment landscape.

Results showed that SRP-5051 30 mg/kg is likely to deliver greater than 10% dystrophin over time with monthly dosing in Duchenne muscular dystrophy.