Neuromuscular

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Thomas Crawford, MD. [LISTEN TIME: 24 minutes]

BridgeBio Pharma met with the FDA to discuss the use of glycosylated α-dystroglycan levels as a surrogate end point for its phase 3 trial FORTIFY investigating BBP-418 and believes there is the potential for an accelerated approval.

The double-blind, placebo-controlled trial assesses change from baseline in the weekly average of daily self-reported worst pain intensity scores after 14 weeks of treatment.

Catch up on any of the neurology news headlines you may have missed over the course of July 2023, compiled all into one place by the NeurologyLive® team.

Results showed that 67% of treated patients in Stage A demonstrated evidence of clinical improvement, indicating that IgG autoantibodies play a significant role in the underlying biology of CIDP.

The trial is expected to include 14 individuals with genetically confirmed limb-girdle muscular dystrophy who will be randomly assigned 1:1 to either AB-1003 or placebo for a year-long treatment period.

The pediatric neurologist at Johns Hopkins Medicine provided commentary on the steps needed to improve treatment optimization in SMA, and the unanswered questions regarding the key biology of the disease.

Here's some of what is coming soon to NeurologyLive® this week.

The pediatric neurologist at Johns Hopkins Medicine provided commentary on the subgroup findings from the phase 4 NURTURE study of nusinersen (Spinraza; Biogen) in presymptomatic spinal muscular atrophy. [WATCH TIME: 3 minutes]

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is sleep disorders.

Neurology News Network for the week ending August 5, 2023. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 4, 2023.

NS-050/NCNP-03, an exon skipping investigational therapy, has been cleared by the FDA for the initiation of a phase 1/2 clinical study for the treatment of Duchenne muscular dystrophy.

The pediatric neurologist at Johns Hopkins Medicine discussed the importance of early diagnosis in SMA, and the notable barriers that come with obtaining disease-modifying therapies. [WATCH TIME: 4 minutes]

Following a successful first patient treatment experience with TSHA-102, an adeno-associated virus vector-based gene therapy for Rett syndrome, Taysha Gene Therapies was recommended to continue with investigating the therapy in a second patient.

Investigators concluded that the growing and testing of motor neurons from patient-derived induced pluripotent stem cells could be clinically used for the prediction of ropinirole’s efficacy as a treatment for patients with ALS.

The pediatric neurologist at Johns Hopkins Medicine provided clarity on the recently published 5-year update of the NURTURE study, and the importance of treating spinal muscular atrophy at its presymptomatic stages.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is epilepsy and seizure disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 28, 2023.

At 19 weeks, eplontersen-treated patients showed substantial and sustained reductions in serum TTR concentration compared with baseline.

Caution is warranted for older patients with Duchenne muscular dystrophy who are receiving gene therapy, according to details of a patient death in a single patient trial.

Here's some of what is coming soon to NeurologyLive® this week.

Neurology News Network for the week ending July 22, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 21, 2023.

Based on the positive findings, the company intends to submit a new drug application to the FDA before the end of 2023, with regulatory filings in additional markets to follow in 2024.

The 52-week, dose-ascending trial will have data read out in the first half of 2024, with long-term safety and efficacy evaluated over a 5-year period following completion of the initial trial.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is the history of the Alzheimer's Association.

After 4 years of treatment, more than 95% of infants on risdiplam maintained the ability to swallow, an aspect of everyday life that is commonly lost within 2 years in patients with SMA.