Neuromuscular

The chief medical officer at ML Bio Solutions, an affiliate of BridgeBio, talked about the importance of awareness for neuromuscular diseases such as Duchenne muscular dystrophy and why a timely diagnosis can make a profound difference in a patient’s life. [WATCH TIME: 3 minutes]

The professor of neurology and translational neuroscientist at Ludwig-Maximillian’s University Munich discussed the reasons behind specific inclusion criteria and outcomes used in the phase 3 ORION study of AMX0035 in progressive supranuclear palsy. [WATCH TIME: 4 minutes]

The double-blind trial will feature 180 patients of various spinal muscular atrophy types who will be followed over a 48-week treatment period.

In a recent multisite analysis of 361 patients with REM sleep behavior disorder, 84% had neurological abnormalities in at least one domain observed.

The adjunct professor of human genetics at Emory University School of Medicine talked about efforts from various communities in neurological disorders, such as Rett syndrome, to develop new measures for research. [WATCH TIME: 3 minutes]

The chief medical officer at ML Bio Solutions, an affiliate of BridgeBio, discussed how promising conversations between researchers and regulatory authorities suggest a potential pathway for accelerated approval for limb-girdle muscular dystrophy type 2I/R9. [WATCH TIME: 3 minutes]

The chief medical officer at ML Bio Solutions, an affiliate of BridgeBio, discussed developing therapies for rare neuromuscular diseases such as limb girdle muscular dystrophy 2I/R9 and the unique challenges that occur in clinical trial settings. [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 Alzheimer and dementia.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 15, 2023.

The adjunct professor of human genetics at Emory University School of Medicine discussed how the Rett Syndrome Behavior Questionnaire has evolved into a versatile tool with a broader scope. [WATCH TIME: 10 minutes]

The chief medical officer at ML Bio Solutions, an affiliate of BridgeBio, talked about the significant progress of investigational agent BBP-418 to treat limb-girdle muscular dystrophy type 2I/R9. [WATCH TIME: 3 minutes]

The Rett Syndrome Behavior Questionnaire’s implementation as global outcome measure in FDA-approved trials was the result of its comprehensive nature since it includes behavior-related symptom questions as well as questions for regular behaviors.

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 stroke.

A composite analysis of motor skills, muscle strength, cognitive ability, daily living skills, and biomarker data showed statistically significant benefits with AMO-02.

Neurology News Network for the week ending September 9, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 8, 2023.

The president of the ANA and the chair of the annual meeting programming committee provided commentary on the advances in neurology and the shift in how clinicians approach and treat diseases once thought to be fatal. [WATCH TIME: 6 minutes]

In honor of World Duchenne Awareness Day, held September 7, 2023, get caught up on some of the latest news in Duchenne muscular dystrophy as the NeurologyLive® team shares some of our data updates.

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

The president of the ANA and the chair of the annual meeting programming committee gave an overview on the various topics and courses the clinical community should home in on at the upcoming annual meeting. [WATCH TIME: 6 minutes]

The director of the ALS Clinic at Allegheny Health Network provided perspective on the advancements in the treatment and management of adult-onset spinal muscular atrophy.

The president of the ANA and the chair of the annual meeting programming committee provided perspective on the upcoming ANA Annual Meeting and what clinicians may get out of the meeting. [WATCH TIME: 7 minutes]

The chief medical officer at Avidity Biosciences talked about how its exon skipping AOC therapies are designed to address the lack of functional dystrophin protein among patients with Duchenne muscular dystrophy to improve muscle function. [WATCH TIME: 4 minutes]