by Peter Ciszewski | Apr 22, 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 an...
by Peter Ciszewski | Apr 18, 2023
The U.S. Food and Drug Administration (FDA) has approved omidubicel (Omisirge), an allogeneic cord blood-based cell therapy to quicken the recovery of neutrophils and reduce the risk of infection. The product is approved for patients 12 years and older with blood...
by Peter Ciszewski | Apr 13, 2023
Paul Orchard, MD, from the University of Minnesota Medical School, provides an overview of metachromatic leukodystrophy (MLD). MLD is a rare lysosomal disorder due to a deficiency in arylsulfatase A (ARSA). It is characterized by the accumulation of...
by Peter Ciszewski | Apr 6, 2023
Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA) talks about the safety of clinical trials. As Dr. Marks makes it clear, clinical trials are monitored but numerous regulatory...
by Peter Ciszewski | Mar 31, 2023
Laura Mamounas PhD, Program Director at the National Institute of Neurological Disorder and Stroke (NINDS) at the NIH in Bethesda, discusses Rett syndrome research at her organization. Rett syndrome is a multisystem disorder that primarily affects girls. Only...
