Neuromuscular

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.

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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.

Recent Foundation for Angelman Syndrome Therapeutics grants funded the hotline as well as work to develop cell line models for rarer genotypes of AS.

Lightheadness, loss of balance, vision changes, and abnormal eye movements led to the discovery of cancer in one specific patient.

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Neurology News Network for the week ending May 1, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending April 30, 2021.

Apic Bio plans to initiate a phase 1/2 clinical trial in late 2021 or early 2022 as a multi-center, 3-part study to evaluate APB-102 in patients with SOD1-ALS mutations.

Formerly known as BIIB067, the antisense oligonucleotide is expected to be available for rapidly progressing patients in mid-July 2021, and for the broader SOD1-ALS population in the fall.

Advances in MRI and MRS techniques and protocols enhance diagnosis of neuromuscular disorders and provide prognostic and intervention efficacy biomarkers.

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Three patients included in the study showed positive trends across both I-RODS and Jamar grip strength following treatment with lenalidomide.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending April 23, 2021.