On Feb. 7, Cambridge, Mass.-based Solid Biosciences announced preliminary results from its phase 1/2 clinical trial, called IGNITE DMD, which is designed to assess the safety and efficacy of its lead drug candidate, SGT-001, in children and adolescents with Duchenne muscular dystrophy (DMD). DMD is caused by a mutation in the dystrophin gene on the X . . .
This week, Arkansas Gov. Asa Hutchinson signed into law a bill, HB 1074, that requires the state to add spinal muscular atrophy (SMA) to its newborn screening panel. SMA is the No. 1 genetic cause of death in infants, affecting approximately 1 in 10,000 babies. MDA was proud to advocate for the addition of SMA . . .
Young adults living with Charcot-Marie-Tooth disease: You can help in a new study. Allison Eveler, a senior occupational therapy major at Elizabethtown College in Pennsylvania, is conducting an undergraduate research study to determine the impacts of CMT on the social participation of young adults. Allison, who herself lives with CMT, recognized a need for research . . .
On Jan. 4, Italian researchers published positive results in the journal SAGE Open Medicine of a Phase 2b clinical trial (MSK-001) evaluating Firdapse (amifampridine phosphate) for treating MuSK antibody positive myasthenia gravis (MuSK-MG). Their results showed the drug to be safe and effective in treating patients with this rare disease. The study was partially funded . . .
On Jan. 4, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Tacoma, Wash.-based Revalesio Corporation’s experimental drug for amyotrophic lateral sclerosis (ALS), RNS60. Fast Track status helps to shorten the time it takes to develop and approve a drug that’s been designed to treat a serious condition and fill an unmet . . .
Researchers at Brainstorm Cell Therapeutics are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a Phase 3 study designed to help researchers evaluate the effects of mesenchymal stromal stem cells secreting neurotrophic factors (MSC-NTF cells) on disease progression in patients with ALS. In ALS, motor neurons degenerate or die, and stop sending . . .
Early identification and treatment for neuromuscular disorders are essential to optimize health outcomes. Newborn screening, which identifies health issues via a blood test taken soon after birth, is essential to ensure that infants born with serious but treatable disorders have the best possible chance at receiving the care and support services they need as early . . .
Researchers at Orphazyme are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the ORARIALS-01 Phase 3 study. The study is designed to help researchers evaluate the effects of arimoclomol on disease progression in patients with ALS. In ALS, motor neurons degenerate or die and stop sending messages to the muscles, which gradually . . .
Researchers at Catabasis are looking for boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study (PolarisDMD). The goal of the study is to evaluate the safety and efficacy of edasalonexent, which researchers believe may slow down progression of skeletal and cardiac muscle disease in DMD by inhibiting NF-kB. NF-kB is a . . .
On Jan. 3, Wave Life Sciences announced that its upcoming Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, has been selected by the U.S. Food and Drug Administration (FDA) for its complex innovative trial designs (CID) pilot program. Wave . . .