Neuromuscular

Key opinion leaders (KOLs) examine the mechanisms of action and other pertinent information regarding FDA-approved treatments for spinal muscular atrophy (SMA), sharing their perspectives on these therapies with a particular focus on the various modes of administration.

The newly added cohort allows for greater opportunity to treat patients earlier, with the hope of impacting disease course and preserving muscle.

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

Vitrisiran demonstrated a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in both the overall population and those who received a monotherapy of vutrisiran without tafamidis.

Neurology News Network. for the week ending June 29, 2024. [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 on epilepsy/Dravet syndrome.

After following patients for more than 10 years, arrhythmias were considered the most important cardiac predictor of survival, with progressive neurologic disability and diabetes mellitus identified as further major contributors.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 28, 2024.

The director of the OhioHealth ALS Clinic and vice chair of the ALS Association Care Services Committee discussed the upcoming ALS Nexus Conference and how themes of the event align with the direction of clinical care. [WATCH TIME: 3 minutes]

Anaya, a young woman with myasthenia gravis, discusses her challenges and triumphs living with a rare disease. She shares her personal story of advocacy and community, finding strength in the disease's impact on her independence and identity.

Additional data of a phase 2 study presented at the 2024 SLEEP Annual Meeting demonstrated that pitolisant significantly improved in secondary end points among patients with myotonic dystrophy type 1.

A neurology specialist offers clinical insights on communicating with patients about the diagnosis of Duchenne muscular dystrophy and discusses the important role of neuromuscular disease specialists.

Emma Ciafaloni, MD, provides an overview of the clinical presentation and diagnosis of Duchenne muscular dystrophy (DMD), highlighting the role of CK testing and the pathophysiology of the disorder.

Clinical experts in spinal muscular atrophy (SMA) address the issue of patient disengagement from healthcare services, which can occur due to a perceived lack of disease progression or stabilization, leading to undetected disease advancement and increased risk to patients, and provide recommendations for encouraging patients to re-engage with medical care.

Key opinion leaders (KOLs) further explore the transition of care from pediatric to adult healthcare systems for patients with spinal muscular atrophy (SMA), emphasizing the essential role of multidisciplinary support in ensuring a successful transition.

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

Neurology News Network. for the week ending June 22, 2024. [WATCH TIME: 4 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 on Alzheimer disease/dementia.

The therapy is the first and only neonatal Fc receptor blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 21, 2024.

The AAV vector-based gene therapy originally received FDA approval for treating ambulatory pediatric patients aged 4 to 5 years with Duchenne under the accelerated approval pathway.

A phase 3 trial showed that over time, Hyqvia is a safe and well-tolerated maintenance treatment for patients with chronic inflammatory demyelinating polyneuropathy.

CNM-Au8, an oral suspension of clean-surfaced, catalytically-active gold nanocrystals, resulted in prolonged survival and reductions in neurofilament light, a prominent biomarker of neuroaxonal damage.

The director at the Foundation for the National Institutes of Health provided commentary on a recently launched partnership aimed at building a repository of ALS data to advance drug development for the disease.

Initial data from a few pediatric and adult patients with Rett syndrome showed that treatment with TSHA-102 resulted in numerous positive enhancements across multiple efficacy measures and clinical domains.