Neuromuscular

Marketed as Elevidys, the gene therapy is approved for ambulatory pediatric patients aged 4 to 5 with Duchenne based on expression of micro-dystrophin.

The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital discussed the benefits opioids have for patients with restless legs syndrome, as well as how they can be safely used as part of treatment strategies. [WATCH TIME: 4 minutes]

Biomarker data at 49 weeks continued to demonstrate that DNL310 enabled rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers.

The basis for the FDA’s decision came from the phase 3 ADAPT-SC study, in which subcutaneous efgartigimod showed a slightly better ability to reduce immunoglobulin compared with its previously approved intravenous formulation.

In one of the first phase 3 trials of primary mitochondrial myopathy, elamipretide failed to meet its primary end points; however, a subgroup of patients with nuclear DNA defects saw improvement in 6-minute walk test, raising further questions.

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 the history of the American Headache Society.

Neurology News Network for the week ending June 17, 2023. [WATCH TIME: 3 minutes]

New data announced by Clene Nanomedicine suggested that the gold nanocrystal therapy reduced plasma neurofilament light chain, which principal investigator Merit Cudkowicz, MD, said, “help advance the design of a phase 3 trial.”

Results from the phase 3 LAVENDER trial investigating trofinetide (Daybue; Acadia), which supported the first FDA-approved treatment for Rett syndrome, were recently published in Nature Medicine.

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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 Parkinson disease and related disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 9, 2023.

The hold comes nearly a month after Health Canada sent PepGen a No Objection Letter to start its phase 2 CONNECT1-EDO51 trial in patients with Duchenne muscular dystrophy.

After discussions with the FDA, the study will assess 2 co-primary end points of change in Rett Syndrome Behavior Questionnaire total score and Clinical Global Impression Improvement Scale score.

The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital provided insight on new updates to the management of restless legs syndrome, including removing dopamine agonists as first line treatments. [WATCH TIME: 8 minutes]

The study did not meet its primary end point of change from baseline in Performance of the Upper Limb 2.0 score at 1 year.

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 the history of the American Academy of Sleep Medicine.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 2, 2023.

With few neuropalliative care services available in the US, the University of Rochester stands as a prime example of how a successful program can transform patient care.

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 disease and dementia.

Over a 72-week treatment period, vatiquinone showed significant impacts on bulbar and upright stability subscales of the modified Friedreich Ataxia Rating Scale score.

Neurology News Network for the week ending May 27, 2023. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 26, 2023.

Fifty-four percent of patients with ALS carried at least one detrimental common variant or repeat expansion, highlighting the clinical impact of gene variants as modifiers in ALS.

Robust, sustained reductions in key pharmacodynamic biomarker were observed; however, this did not lead to functional benefits on any of the scales used in the trial.

Submitted under the accelerated approval pathway, if approved, SRP-9001 would become the first marketed gene therapy specific for Duchenne muscular dystrophy.

The consultant and founder of Accelerating NeuroVentures talked about the pridopidine arm from the HEALEY ALS Platform trial, which showed significant benefits in speech measures. [WATCH TIME: 5 minutes]