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...
by James Radke, PhD | Apr 17, 2023
Nils Confer, Vice President, Medical and Patient Strategies at Aceragen, provides an overview of Farber disease. Farber disease is a lysosomal disorder due to mutations in the ASAH1 gene. It leads to reduced activity of acid ceramidase. This deficiency results...
by James Radke, PhD and Madaline Spencer | Apr 13, 2023
Samiah Al-Zaidy, MD, Vice President of Clinical Development and Lead on the Passage Bio GM1 Gangliosidosis (GM1) Program, discusses the updated data from Imagine-1 evaluating PBGM01 in GM1, which was presented at WORLDSymposium 2023. Background of GM1 GM1 is an...
