by Madaline Spencer | Mar 19, 2026
Gabriel Brooks, MD, Chief Medical Officer at Solid Biosciences, and Russell Lonser, MD, of The Ohio State University Wexner Medical Center, discuss SGT-212 and the FALCON clinical trial for patients with Friedreich’s ataxia. Friedreich’s ataxia is a...
by Madaline Spencer | Mar 18, 2026
The US Food and Drug Administration (FDA) has approved Wellcovorin (leucovorin calcium) tablets for the treatment of adult and pediatric patients with cerebral folate deficiency (CFD) with confirmed variants in the folate receptor 1 gene (CFD-FOLR1). The drug was...
by Madaline Spencer | Mar 18, 2026
Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses hematopoietic stem cell gene therapy (HSCGT) in patients with mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo syndrome type A). MPS IIIA is a genetic...
by Madaline Spencer and Joe Haddad | Mar 17, 2026
Amel Karaa, MD, Genetics and General Metabolism, Director of the Mitochondrial Disease Program at Massachusetts General Hospital, discusses the PRIZM clinical trial of zagociguat in patients with MELAS. MELAS, or Mitochondrial Encephalomyopathy, Lactic...
by Madaline Spencer | Mar 16, 2026
Rachele Berria, MD, PhD, Senior Vice President, Head of Global Medical Affairs at Chiesi Rare Diseases, gives an overview of the company’s highlights at WORLDSymposia 2026. According to Dr. Berria, the biggest unmet needs in lysosomal storage disorders...
