by Madaline Spencer | Sep 16, 2024
Ataxia telangiectasia (A-T) is a rare primary immunodeficiency that affects the nervous system, the immune system, and many other parts of the body. The condition is typically characterized by early childhood onset of cerebellar ataxia, oculomotor apraxia,...
by Madaline Spencer | Sep 13, 2024
The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to NS-050/NCNP-03 for the treatment of Duchenne muscular dystrophy (DMD). DMD is a rare disease affecting the muscles, leading to muscle wasting that gets worse over time. It is...
by Madaline Spencer | Sep 12, 2024
Richard Bedlack, MD, PhD, Stewart, Hughes and Wendt Distinguished Professor of ALS at Duke University, discusses a study identifying genetic associations with amyotrophic lateral sclerosis (ALS) reversals. ALS, also referred to as “Lou...
by Madaline Spencer | Sep 11, 2024
Ben Zimmer, CEO of Priovant Therapeutics, discusses the VALOR clinical trial testing investigational therapy for dermatomyositis treatment, brepocitinib. Dermatomyositis is a rare autoimmune condition that causes skin changes and muscle weakness....
by Madaline Spencer | Sep 6, 2024
Corey Cutler, MD, Director of Adult Stem Cell Transplantation Program at Dana-Farber Cancer Institute in Boston and a Professor of Medicine at Harvard Medical School, discusses the approval of Niktimvo (axatilimab) for the treatment of chronic graft versus host...
