Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 29, 2023.

The professor of neurology and translational neuroscientist at Ludwig-Maximillian’s University Munich detailed the logic behind the ORION study, a new trial assessing AMX0035, an approved therapy for ALS, in patients with progressive supranuclear palsy. [WATCH TIME: 4 minutes]

In a phase 3 setting, the 2-component therapy showed positive trends or clinically meaningful improvements on motor and respiratory functions compared with previously approved enzyme replacement therapy.

As part of our monthly clinician spotlight, NeurologyLive® highlighted neueomuscular expert Peter Kang, MD, director of the Paul and Sheila Wellstone Muscular Dystrophy Center and professor of neurology at the University of Minnesota.

After deliberating, the FDA committee expressed concerns about the mechanism of action of NurOwn, manufacturing details, and consistency of data.

Coya Therapeutics expects to release data from the biomarker analyses of COYA 302 and publish the full results from the trial in a peer-reviewed journal in the second half of the year.

Additional biomarker findings from up to 2 years of DNL310 treatment suggest positive changes in adaptive behavior and cognition as well as improvement in auditory function among pediatric patients with Hunter syndrome.

In an integrated meta-analysis of the HEALEY ALS platform trial and RESCUE-ALS trial, 30mg of CNM-Au8 resulted in 59% decreased risk of mortality among participants with ALS compared with PRO-ACT matched placebo over long-term follow-up.

The director of the Duke ALS Clinic at Duke Health provided perspective on the issues with getting newly approved ALS therapies to patients who need them the most. [WATCH TIME: 4 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 headache and migraine.

In the study, patients with ALS will be asked to complete questionnaires, a physical examination, and undergo neurostimulation site visits 3 times per week for 4 weeks.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 22, 2023.

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]