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...
by Madaline Spencer | Mar 15, 2024
Roberto Giugliani, MD, PhD, discusses the RAINBOW clinical trial testing the tolerability and safety of nizubaglustat for Niemann-Pick type C and GM2 gangliosidosis. Niemann-Pick Type C Niemann-Pick disease is a genetic metabolic disorder in which...
by Madaline Spencer | Mar 13, 2024
Aki Ko, Chief Executive Officer and co-founder of Elixirgen Therapeutics, discusses treatment advances in telomere biology disorders. Telomere biology disorders are a group of conditions that occur when patients have premature or faster shortening of...
