Researchers at PTC Therapeutics are looking for individuals with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy of deflazacort (Emflaza), which researchers hope may reduce inflammation in muscles and potentially lead to improved muscle strength and motor function. Participants will . . .
Researchers at Mitsubishi Tanabe Pharma Development America are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a phase 3 study to evaluate the safety of oral edaravone (Radicava). Intravenous edaravone is an FDA-approved treatment for ALS shown to slow the decline of physical function. Edaravone is thought to act as a free . . .
On Jan. 22, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 FIREFISH clinical trial assessing risdiplam in infants age 1 to 7 months with spinal muscular atrophy (SMA) type 1. The company announced that the study met its primary endpoint, which was a proportion of infants . . .
Researchers at Scholar Rock are looking for individuals with spinal muscular atrophy (SMA) type 3 to participate in a phase 2 study. The goal of the study is to evaluate the safety and efficacy of SRK-015, which researchers hope may increase muscle mass and potentially improve muscle strength and motor function. The total study duration . . .
Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its primary endpoint, which was to assess the safety and tolerability of ATL1102 in patients with DMD who are non-ambulatory (unable to walk independently). After six . . .
AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich’s ataxia (FA), was awarded MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio’s phase 2 clinical trial of a gene-replacement therapy for the disease. MVP is MDA’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and . . .
On Dec. 13, the US Food and Drug Administration (FDA) approved expanded labeling for ITF Pharma’s Tiglutik to include administration via percutaneous endoscopic gastrostomy (PEG) tube for the treatment of amyotrophic lateral sclerosis (ALS). The expanded approval for use with PEG feeding tubes will enable patients who not only have difficulty swallowing but also require . . .
Yesterday, Mallinckrodt Pharmaceuticals released a statement to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full statement follows. After careful consideration and despite engaging in diligent efforts to research, develop, and commercialize MNK-1411, Mallinckrodt has made the decision to terminate study MNK14112096 (BRAVE). The study was designed to . . .
On Dec. 17, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced positive results from the phase 2 (CENTAUR) trial assessing treatment with the company’s investigational therapy, AMX0035, in patients with amyotrophic lateral sclerosis (ALS). The study met its primary endpoint, showing a statistically significant slowing of . . .
Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding updates on a serious adverse event (SAE) experienced by one child dosed last November and new biomarker data from the company’s IGNITE DMD clinical trial. The trial is still on hold pending review by the US Food and Drug Administration (FDA). The full . . .