On Oct. 16, Amylyx announced an update from its CENTAUR trial evaluating the effects of the investigational drug AMX0035 (a combination of the small molecules sodium phenylbutyrate and taurursodiol) on overall survival in people with amyotrophic lateral sclerosis (ALS). The current study followed each participant in the CENTAUR trial for up to three years from . . .
On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .
Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .
On Aug. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third disease-modifying therapy approved to treat SMA, the leading genetic cause of infant death. The drug is an orally administered liquid, the first medicine that could . . .
On July 18, MDA is hosting a free virtual Engage Limb-Girdle Muscular Dystrophy Symposium, a half-day educational event featuring key experts who will speak on topics including the latest in genetics and research in limb-girdle muscular dystrophy (LGMD). MDA has a longstanding commitment to finding treatments for the more than 30 subtypes of LGMD; since . . .
On June 15, Sarepta Therapeutics announced positive results from its phase 1/2a study of SRP-9001 gene therapy to treat Duchenne muscular dystrophy (DMD). Data from four patients indicated that a single intravenous infusion of SRP-9001 was safe and well tolerated, with no serious adverse events. Additionally, all participants demonstrated improvements across multiple efficacy-related endpoints, including . . .
On June 15, Momenta Pharmaceuticals announced positive results from its phase 2 Vivacity-MG trial assessing treatment with the company’s investigational therapeutic nipocalimab (M281) in patients with generalized myasthenia gravis (gMG). Preliminary data indicated that treatment with nipocalimab, using four different dosing protocols over an eight-week period, resulted in improvements in patients’ Myasthenia Gravis Activities of . . .
Stephan Züchner, MD, PhD, professor for Human Genetics and Neurology, Chair of the Human Genetics Department, and codirector of the John P. Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine, recently discovered mutations in the sorbitol dehydrogenase gene (SORD) that cause a recessive, axonal form of Charcot-Marie-Tooth disease (CMT) . . .
On June 8, Sarepta Therapeutics announced positive results from its first study in human patients of SRP-9003, a gene therapy designed to treat limb-girdle muscular dystrophy (LGMD) type 2E. Data from low-dose and high-dose patient cohorts indicated that a single intravenous infusion of SRP-9003 was well tolerated, with only one serious adverse event occurring in . . .
On June 2, ReveraGen BioPharma announced the completion of two-and-a-half years of vamorolone treatment in 41 boys with Duchenne muscular dystrophy (DMD). The long-term data from this study indicated that daily oral administration of vamorolone, even at its highest tested dose, was safe and well tolerated with no serious adverse events. Preliminary data have shown . . .