Neuromuscular

The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.

The first patient enrolled into the phase 1/2a ArthemiR trial assessing ATX-01 myotonic dystrophy type 1 is expected in the second quarter of 2024.

Experts in neurology highlight taking age and quality of life into consideration when approaching treatment of MG, as well as comment on the standard of care for patients.

Expert neurologists discuss the factors that guide treatment selection in MG, shifting the framework to take patient preferences into account.

The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles discussed the hesitancy to enroll children with neurodevelopmental issues in early intervention trials without proven products or markers. [WATCH TIME: 5 minutes]

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 on limb girdle muscular dystrophy.

The phase 1 study, dubbed PRO-101, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in both patients with ALS and healthy volunteers.

Neurology News Network. for the week ending April 6, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending Aprl 5, 2024.

The executive medical lead at Italfarmaco SpA expressed his confidence in givinostat to mitigate the progression of Duchenne muscular dystrophy, emphasizing its multifactorial mechanism of action. [WATCH TIME: 5 minutes]

Amylyx Pharmaceuticals’ AMX0035, despite its safety profile, did not show significant efficacy among patients with amyotrophic lateral sclerosis in the phase 3 PHOENIX trial.

TUDCA, a hydrophilic bile acid that is normally produced endogenously in humans in the liver, failed to slow ALS disease progression in comaprison with placebo.

The executive medical lead at Italfarmaco SpA talked about data from both animal models and clinical trials supporting the efficacity of givinostat in patients with Duchenne muscular dystrophy at MDA 2024. [WATCH TIME: 5 minutes]

The associate professor of neurology at the University of Naples discussed a recently enrolled study assessing the use of dimethyl fumarate, an approved therapy for relapsing MS and psoriasis, in patients with Friedreich ataxia.

The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles provided insight on the barriers to prevent neurodevelopmental disorders and the lack of currently validated biomarkers. [WATCH TIME: 4 minutes]

Ali Habib, MD, comments on the prevalence of MG in older patients and the increasing rate of hospitalization and mortality within this group.

Beth Stein, MD, reviews critical steps towards diagnosing MG, taking a look at the different types of reliable testing for the disease.

At some dose levels, the treatment appeared to slow the rate of motor function decline and regenerate sensory nerves in patients with giant axonal neuropathy.

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 multiple sclerosis and MS mimics.

A recent study presented key predictors of survival in patients living with Friedreich ataxia, which led to the creation of a prognostic model for disease management.

Neurology News Network. for the week ending March 30, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 29, 2024.

The associate professor of neurology at the University of Naples provided context on the mechanism of action for dimethyl fumarate and why there’s belief it can benefit patients with Friedreich ataxia. [WATCH TIME: 4 minutes]

The chief executive officer at Elixirgen Therapeutics discussed the company’s mRNA as a promising therapeutic avenue for Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

Over the 24-week treatment period, satralizumab demonstrated a well tolerated safety profile that was consistent with its previous indication.

The professor of neurology at Vanderbilt University Medical Center discussed advocating for reinstating personalized resources, expanding genetic testing, and improving transitional care in muscular dystrophy. [WATCH TIME: 5 minutes]

The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]

The neuromuscular specialist at the Neurology & Neuromuscular Care Center discussed the pressing need to address the transition of care from pediatric to adult healthcare systems in Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]