by Madaline Spencer | Mar 23, 2026
Geraldine Bliss, Co-Founder and President of CureSHANK, and Jenny Graham Beeson, Board Member for CureSHANK, discuss clinical research in Phelan-McDermid syndrome (PMS) and the Start Genetic initiative. PMS is a chromosome disorder caused by deletions at...
by Madaline Spencer | Mar 20, 2026
Peng Lu, MD, PhD, Chief Medical Officer at Pharvaris, and Wim Souverijns, PhD, Chief Commercial Officer at Pharvaris, discuss the RAPIDe-3 clinical trial of deucrictibant for patients with hereditary angioedema (HAE). Hereditary angioedema (HAE) is a...
by Madaline Spencer | Mar 19, 2026
Gabriel Brooks, MD, Chief Medical Officer at Solid Biosciences, and Russell Lonser, MD, of The Ohio State University Wexner Medical Center, discuss SGT-212 and the FALCON clinical trial for patients with Friedreich’s ataxia. Friedreich’s ataxia is a...
by Madaline Spencer | Mar 18, 2026
The US Food and Drug Administration (FDA) has approved Wellcovorin (leucovorin calcium) tablets for the treatment of adult and pediatric patients with cerebral folate deficiency (CFD) with confirmed variants in the folate receptor 1 gene (CFD-FOLR1). The drug was...
by Madaline Spencer | Mar 18, 2026
Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses hematopoietic stem cell gene therapy (HSCGT) in patients with mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo syndrome type A). MPS IIIA is a genetic...
