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....
by James Radke, PhD | Apr 26, 2023
The U.S. Food and Drug Administration (FDA) has approved tofersen (Qalsody) to treat individuals with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Tofersen is an antisense oligonucleotide that...
by James Radke, PhD and Madaline Spencer | Apr 24, 2023
Alex Dorenbaum, MD, Chief Medical Officer of Reneo Pharmaceuticals, gives an overview and update of the STRIDE study of mavodelpar (REN001) in primary mitochondrial myopathies (PMM). What Are PMMs? PMMs are a group of rare metabolic disorders caused by...
by James Radke, PhD and Craig McCabe | Apr 19, 2023
MSD is a rare genetic disease due to mutations in the SUMF1 gene and absences of SUMF1 leads to the buildup of glycosaminoglycans in cells and organs. MSD is a progressive disease. There is no treatment currently available that targets the pathophysiology of this...
