Neuromuscular

The majority of patients with ALS lack a monogenic mutation, with only 15-20% of patients having a disease that is driven and controlled by a single gene.

Neurology News Network for the week ending August 12, 2023. [WATCH TIME: 3 minutes]

Since it was first discovered in the early 1900s, the therapeutic approaches to treating myasthenia gravis have changed significantly, with several proven treatment options.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 11, 2023.

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.