by Madaline Spencer | Sep 18, 2024
A new gene therapy for X-linked retinoschisis (XLRS) has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). This rare genetic disorder affects the retina, causing vision loss primarily in males. The designation marks a crucial step...
by Madaline Spencer | Sep 17, 2024
Bruce Leuchter, MD, co-founder and CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses topline results from clinical trial testing investigational radiprodil for GRIN-related disorders. GRIN-related neurodevelopmental disorders are a family of...
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...
