Neuromuscular Disease News

The FDA has granted orphan drug designation to DTx-1252, an investigational small interfering RNA therapeutic to treat Charcot-Marie-Tooth disease type 1A.

Apellis Pharmaceuticals Inc. announced that its phase 2 MERIDIAN study investigating systemic pegcetacoplan as a treatment for ALS failed to meet efficacy measures of overall function, survival, lung function and muscle strength.

Topline results from a phase 1b/2a study evaluating an investigational therapy for C9orf72-associated ALS and frontotemporal dementia revealed no clinical benefit after 24 weeks of treatment compared with placebo.

BOSTON — Long-term treatment with Vyvgart was safe, well-tolerated and effective in reducing immunoglobulin G antibody levels and improving clinical outcomes in patients with generalized myasthenia gravis.

The American Academy of Neurology has elected Carlayne E. Jackson, MD, FAAN, a neurologist, researcher and professor of neurology and otolaryngology at the University of Texas Health Science Center at San Antonio, as its 38th president.

The FDA has approved Qalsody, an antisense oligonucleotide, to treat individuals with ALS associated with a mutation in the superoxide dismutase 1 gene.

The FDA has granted fast track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping, Avidity Biosciences announced in a press release.

BOSTON — Eculizumab significantly reduced disease severity and improved quality of life at 26 weeks in adolescents with refractory generalized myasthenia gravis, according to a presenter at the American Academy of Neurology annual meeting.

The FDA has approved Prospera, a spinal cord stimulation system to treat those with chronic intractable pain in the upper body and limbs.

Continuous treatment with avalglucosidase alfa, or after a switch from alglucosidase alfa, maintained respiratory function and functional endurance in patients with late-onset Pompe disease, with no new safety concerns reported.