Ryan Colburn is a rare disease patient with a professional background in engineering and operations management, spending portions of his career working on racecars, airplanes, and rockets. Diagnosed with Pompe disease in 2015, he has spent the time since learning about rare disease topics including research, advocacy, and drug development to better understand how to . . .
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 . . .
Today, MDA announced new appointments to its board of directors. Steven J. Farella will now serve as chairman and Donald S. Wood, PhD, will serve as vice chairman of the MDA Board of Directors. “We are very pleased to share that both Steven Farella and Dr. Donald Wood have accepted their new positions on the . . .
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 . . .
On Nov. 19, Santhera Pharmaceuticals announced publication of long-term data from its SYROS study showing a reduction of decline in lung function in people with Duchenne muscular dystrophy (DMD) who were treated with idebenone, the company’s experimental therapy for respiratory symptoms associated with DMD. The data from the retrospective study was published in the journal Neuromuscular . . .
Today, Wave Life Sciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program for suvodirsen, its therapy for patients amenable to exon 51 skipping, and its therapy for patients amenable to exon 53 skipping. The full letter follows. Dear Duchenne community: We have a disappointing update . . .
On Dec. 12, the US Food and Drug Administration (FDA) granted accelerated approval to golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the second exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .