by Madaline Spencer | Mar 22, 2024
The U.S. Food and Drug Administration (FDA) has approved givinostat for the treatment of patients with all genetic variants of Duchenne muscular dystrophy (DMD). DMD is caused by genetic changes in the DMD gene that leads to a reduction in functioning levels of the...
by Madaline Spencer | Mar 21, 2024
Trevor Baglin, MD, PhD, Vice President of Centessa Pharmaceuticals UK, discusses SerpinPC, an investigational drug candidate, for the potential treatment of severe hemophilia. Hemophilia is a bleeding disorder that slows the blood clotting process....
by Madaline Spencer | Mar 21, 2024
Matt Trudeau, head of ITF Therapeutics, discusses givinostat, an investigational therapy for Duchenne muscular dystrophy (DMD). DMD is a rare neuromuscular disease caused by genetic variants in the DMD gene. The disease primarily affects the muscles,...
by Madaline Spencer | Mar 19, 2024
The U.S. Food and Drug Administration (FDA) has approved Lenmeldy (atidarsagene autotemcel) for the treatment of children with metachromatic leukodystrophy (MLD). The treatment is the first gene therapy to be approved by the FDA for children with this indication....
by Madaline Spencer | Mar 19, 2024
Ozlem Goker-Alpan, MD, Founder and CMO of Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), discusses newborn screening for lysosomal disorders. Newborn screening plays a crucial role in the diagnosis of rare diseases. Waiting for the...
