The NeurologyLive neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
March 08, 2021
Article
Here's what is coming soon to NeurologyLive.
March 07, 2021
Article
The 2-stage study is an open-label, 2-stage clinical trial designed to evaluate safety and dose-escalation (stage 1) and safety and efficacy (stage 2) of surgically delivered AXO-AAV-GM2.
March 05, 2021
Article
Take 5 minutes to catch up on NeurologyLive's highlights from the week ending March 5, 2021.
March 04, 2021
Video
The director of research analytics at Cure SMA discussed her research on the economic burden and costs patients with spinal muscular atrophy and their caregivers face.
March 03, 2021
Article
Protalix BioTherapeutics intends to report final data on the agent, also known as PRX-102 in the second half of 2021, as well as present those findings at an appropriate medical conference.
March 02, 2021
Article
The FIREFISH clinical trial by PTC Therapeutics is 1 of several studies underway evaluating risdiplam in SMA.
March 02, 2021
Article
The recommendation stems from recently published topline data which demonstrated a clinically meaningful treatment response compared to placebo but failed to meet statistical significance.
March 01, 2021
Article
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February 26, 2021
Article
Take 5 minutes to catch up on NeurologyLive's highlights from the week ending February 26, 2021.
February 25, 2021
Article
The antisense oligonucleotide casimersen will be branded as Amondys 45 and is currently being assessed in the ESSENCE phase 3 clinical trial (NCT02500381).
February 25, 2021
Article
Advancements in gene therapy approaches have opened the door for a glimpse into the future of care for a number of diseases.
February 23, 2021
Video
The assistant professor of pediatric neurology at the Washington University in St. Louis spoke to the insights of a recent survey she and colleagues conducted.
February 22, 2021
Video
The director of the Massachusetts General Hospital ALS Care Center detailed the role genetic backgrounds can have on outcomes within ALS clinical trials.
February 22, 2021
Article
Here's what is coming soon to NeurologyLive.
February 20, 2021
Video
The director of the Massachusetts General Hospital ALS Care Center discussed some of the biggest challenges for patients with ALS and what should be prioritized in research.
February 19, 2021
Article
Take 5 minutes to catch up on NeurologyLive's highlights from the week ending February 19, 2021.
February 19, 2021
Article
Overall, variants within known ALS-linked genes are of potential clinical importance for 42% of patients, according to the study authors.
February 18, 2021
Article
Scholz was recognized for her application of advanced genetic techniques to the study of neurodegenerative disorders, including dementia with Lewy bodies, multiple system atrophy, frontotemporal dementia, progressive supranuclear palsy, and corticobasal degeneration.
February 17, 2021
Article
The DMD treatment pipeline boasts a number of therapeutics with varying mechanisms of action and delivery systems, pointing to a promising future for treatment.
February 15, 2021
Article
Researchers found that 93% of patients with AS exhibited dystonia in their limbs, mouth, neck, or trunk.