by Peter Ciszewski | May 19, 2023
Today, the U.S. Food and Drug Administration (FDA) approved a gene therapy for persons with dystrophic epidermolysis bullosa (DEB). More specifically, it is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy (beremagene geperpavec or Vyjuvek) approved to...
by Peter Ciszewski | May 3, 2023
Aimee Donald, MBChB, PhD, Pediatrician at Royal Manchester Children’s Hospital, and Professor at the University of Manchester, discusses Gaucher disease type 2. Gaucher disease is a rare lysosomal disorder due to reduced levels of glucocerebrosidase that...
by Peter Ciszewski | May 1, 2023
U.S. Food and Drug Administration (FDA) has approved somapacitan ( Sogroya) to treat children aged 2.5 years and older with growth hormone deficiency. The orphan drug was previously approved to treat adults with growth hormone deficiency. Somapacitan is administered...
by Peter Ciszewski | Apr 28, 2023
Akram Khan, MD, FCCP, Associate Professor of Pulmonary, Allergy and Critical Care Medicine at Oregon Health & Science University, discusses the new clinical guidelines on respiratory management of patients with neuromuscular weakness, which was recently...
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...
