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 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 . . .
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 . . .
Drug companies are developing more therapies for rare disease patients — and doing it faster — than ever before. But when it comes to enrolling minorities in clinical trials, the US track record is lagging behind. That subject was the focus of a March 15 session during the 2022 MDA Clinical & Scientific Conference in . . .
Researchers at Alexion Pharmaceuticals are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ALXN2050. ALXN2050 is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including gMG. People . . .
Researchers at Alexion Pharmaceuticals are seeking adults living with dermatomyositis to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ravulizumab (Ultomiris). Ravulizumab is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including dermatomyositis. Treatment with ravulizumab . . .
Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care. Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .