Myasthenia gravis (MG) is a chronic neuromuscular disease characterized by muscle weakness that worsens after activity and improves after rest. MG is caused by an autoimmune reaction in which the body’s immune system attacks its own tissues, interrupting the connection between nerves and muscles (the neuromuscular junction). MG can occur at any age and affects . . .
On May 31, Eledon announced positive topline results from its Phase 2a clinical trial of the investigational therapy tegoprubart (formerly AT-1501) for treatment of amyotrophic lateral sclerosis (ALS). The primary endpoints of the study, assessment of safety and tolerability, demonstrated an encouraging safety profile that supports further investigation. Secondary endpoints showed that the drug engaged . . .
Friedreich’s ataxia (FRDA) is an inherited neuromuscular disease that primarily impacts the nervous system and heart and affects about one in 50,000 people worldwide. FRDA typically appears in people before the age of 25 years and is characterized by a slow, progressive loss of limb coordination (ataxia) and effects on speech and swallowing. While there . . .
Researchers at the Barrow Neurological Institute (BNI) and Emory University are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in the Track-ALS research study to assess whether measurements collected at home, using digital applications, are dependable and can help with tracking disease progression. Study participants will be asked to perform various at-home tests at . . .
Researchers at Cytokinetics, Inc. are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in the phase 3 COURAGE-ALS clinical trial to evaluate the safety and effectiveness of the investigational drug. In ALS, motor neurons (nerve cells that control muscle cells) are gradually lost, causing the skeletal muscles they control to become weak and nonfunctional. . . .
On May 12, the US Food and Drug Administration (FDA) granted approval to an oral suspension of edaravone (RADICAVA ORS) for the treatment of amyotrophic lateral sclerosis (ALS). To date, RADICAVA ORS is the sixth disease modifying drug approved to treat ALS. RADICAVA ORS will be made available in the United States (US) and marketed . . .
Researchers at the University of California, Davis Neuromuscular Research Lab are seeking individuals with or without neuromuscular disease (ages 2 years or older) to participate in a research study to test the feasibility of using a wearable gait sensor to collect data as part of a new outcome measure for use in future clinical trials. This . . .
On April 28, the US Food and Drug Administration (FDA) granted approval to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United . . .
Inclusion body myositis (IBM) is a rare, progressive muscle disease characterized by chronic muscle inflammation and weakness. It is estimated that approximately 20,000 people in the United States (US) have IBM, though the exact prevalence is unknown. IBM usually develops after age 50 and is more likely to affect men than women. The disease progresses . . .
On April 14, Edgewise Therapeutics announced the initiation of an Edgewise-funded observational trial in individuals with Becker muscular dystrophy (BMD). The goal of trial is to understand the progression of disease in individuals with BMD as assessed by functional measures and imaging endpoints. BMD is a genetic disease caused by a mutation in the dystrophin . . .