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...
by Madaline Spencer | Mar 13, 2026
Amy Shapiro, MD, a pediatric hematologist and the Medical Director and CEO of the Indiana Hemophilia and Thrombosis Center in Indianapolis, provides CheckRare an overview on type 1 plasminogen deficiency (PLGD-1). « Prev1 / 1Next »Type 1 Plasminogen...
by Madaline Spencer and Joe Haddad | Mar 13, 2026
Kristin McKay, President and Executive Director of Project Alive, discusses the real-world perspective on daily challenges of living with mucopolysaccharides II (MPS II). MPS II is an inherited disorder of carbohydrate metabolism that occurs almost exclusively...
