by Peter Ciszewski | Mar 24, 2018
Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy — one of nine major types of muscular dystrophy that affects males — live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of...
by Peter Ciszewski | Mar 24, 2018
A Phase 2 clinical trial of ATL1102, Antisense Therapeutics’ investigational therapy for Duchenne muscular dystrophy (DMD), recently received authorization to proceed from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee. ATL1102 inhibits...
by Peter Ciszewski | Mar 24, 2018
Solid Biosciences announced that the U.S. Food and Drug Administration placed a clinical hold on a Phase 1/2 trial testing SGT-001, a dystrophin gene therapy intended to treat Duchenne muscular dystrophy (DMD), after the first patient dosed was hospitalized with a...
by Peter Ciszewski | Mar 20, 2018
Jeff Marrazzo is Co-Founder and CEO of Spark Therapeutics. In this video, he discusses his company’s investigational gene therapy programs focused on Hemophilia. Hemophilia is most typically a rare genetic bleeding disorder that causes a delay in clot formation...
by Peter Ciszewski | Mar 19, 2018
Thijs Spoor of AzurRx Provides an Overview of Cystic Fibrosis Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An estimated 30,000 children and adults in the United States (70,000 worldwide) have CF. In...
