by Peter Ciszewski | Mar 7, 2022
Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, discusses RGX-111, an investigational gene therapy for mucopolysaccharidosis type I (MPS I). Data from...
by Peter Ciszewski | Mar 5, 2022
Steve Pakola, MD, Chief Medical Officer for Regenxbio, discusses data from the ongoing gene therapy trials in children with mucopolysaccharidosis type I (MPS I) and mucopolysaccharidosis type II (MPS II). The data was presented at WORLDSymposium 2022. MPS I is...
by Peter Ciszewski | Mar 4, 2022
Russ Jelsema, MD, Senior Medical Director at Natera, discusses the results of the SMART study, which evaluated the performance of Single Nucleotide Polymorphism (SNP)-based Non Invasive Prenatal Testing (NIPT) for 22q11.2 deletion syndrome (22q) in a cohort of...
by Peter Ciszewski | Mar 3, 2022
Jonathan Wall, PhD, Director of the University of Tennessee Graduate School of Medicine’s Amyloidosis and Cancer Theranostics Program and Co-founder & Interim Chief Scientific Officer at Attralus, explains why immunoglobulin light chain-associated (AL)...
by Peter Ciszewski | Mar 3, 2022
Giles Campion, MD, Head of R&D at Silence Therapeutics, discuss SLN124, a gene silencing therapy in development for the treatment of thalassemia. As Dr. Campion explains, thalassemia is an inherited blood disorder that reduces the production of functional...
