Neuromuscular Disorders

Latest News

Ultomiris Approved for Generalized Myasthenia Gravis

The approval was based on data from a phase 3 study that included 175 adults with generalized myasthenia gravis who were not previously treated with a complement inhibitor.

Age at Loss of Ambulation Tied to Peak Motor Skills in Patients With DMD

Researchers sought to evaluate the association between functional test scores recorded at peak age for male children with DMD, and the age of those children at LOA.

CAP-1002 Shows Promise for Late-Stage Duchenne Muscular Dystrophy in HOPE-2 Trial

Researchers sought to evaluate the safety and efficacy of repeated intravenous cardiosphere-derived cell therapy in patients with late-stage Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy: Time Interval Unchanged from Symptoms to Diagnosis

Researchers sought to assess if there were any improvements in the time interval from initial symptoms to diagnosis in a follow-up investigation of patients with DMD born after 2000.

FDA Advisory Panel Narrowly Votes Against New ALS Drug

In a close vote, an advisory panel for the US Food and Drug Administration decided not to recommend the approval of an experimental drug for amyotrophic lateral sclerosis (ALS).

FDA Panel Votes on Fixed-Dose Combination Therapy for ALS

AMX0035 is an oral, fixed-dose coformulation of sodium phenylbutyrate and taurursodiol, also known as ursodoxicoltaurine.

BMI-Z Scores in Patients With Duchenne Muscular Dystrophy Before Ambulation Loss

Researchers sought to analyze weight changes during Duchenne muscular dystrophy and the presence of any associations with ambulatory status, caloric intake, or corticosteroid use.

Low-Grade Gliomas With Elevated DMD Expression Tied to Shorter Survival

Researchers sought to characterize the association between the expression of the Duchenne muscular dystrophy gene and low-grade glioma survival outcomes.

Specific Muscular Dystrophy Mutational Sites Not Associated With Epilepsy Risk

In a systematic review and meta-analysis, researchers evaluated the evidence of a relationship between muscular dystrophy mutational sites and epilepsy risk.

Ravulizumab Sustains Improvement in Patients With Generalized Myasthenia Gravis

Researchers reported results from an interim analysis of an ongoing open-label extension to study the safety and efficacy of adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.