by James Radke, PhD | May 9, 2023
Giacomo Chiesi, Head of Chiesi Global Rare Diseases, discusses velmanase alfa (Lamzede), which was recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of alpha mannosidosis. Alpha mannosidosis is a multisystemic, lysosomal disorder...
by James Radke, PhD and Craig McCabe | May 8, 2023
Brenna Bentley , MS, GCG, genetic counselor and patient research coordinator for the United MSD Foundation lists some of the more common symptoms observed in children with multiple sulfatase deficiency (MSD). MSD is a rare genetic disease due to mutations in...
by Craig McCabe and James Radke, PhD | May 8, 2023
Gerard Vockley, MD, PhD, Professor of Pediatrics and Human Genetics at the University of Pittsburgh, provides an overview of galactosemia. Galactosemia is a rare, metabolic disorder that affects an individual’s ability to metabolize galactose, which is...
by James Radke, PhD | May 2, 2023
Jordi Diaz-Manera, MD, PhD, Professor of Neuromuscular Diseases at Newcastle University discusses the latest results from an ongoing phase1/2 clinical trial testing gene therapy for late-onset Pompe disease. Pompe disease is a lysosomal disorder due to...
by James Radke, PhD | Apr 27, 2023
Raphael Schiffmann, MD, of 4D Molecular Therapeutics. Schiffmann, discusses why the US Food and Drug Administration (FDA) put a hold on a phase 1 / 2 clinical trial testing 4D-310, a gene therapy in development that targets the heart in Fabry disease....
