by Peter Ciszewski | May 20, 2022
Priya Kishnani, MD, Chief, Division of Medical Genetics and Professor of Pediatrics at the Duke University School of Medicine, discusses the need for a team approach to care for Pompe disease. Pompe disease is a rare inherited lysosomal storage disorder...
by Peter Ciszewski | May 16, 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). Positive Data from Regenxbio’s Gene Therapy...
by Peter Ciszewski | May 14, 2022
The U.S. Food and Drug Administration (FDA) has approved an oral form of edaravone (Radicava ORS), for the treatment of amyotrophic lateral sclerosis (ALS), a rapid neurodegenerative disease. An intravenous (IV) formulation of edaravone was approved in 2017. ALS, also...
by Peter Ciszewski | May 12, 2022
Sherif El-Harazi, MD, Ophthalmologist and Medical Director and Founder of Lugene Eye Institute and Global Research Management, discusses cyclosporine ophthalmic emulsion 0.1% eye drops (Verkazia) which is now available in the United States for the treatment of...
by Peter Ciszewski | May 10, 2022
Giacomo Chiesi, MBA, Head of Chiesi Global Rare Diseases, discusses a white paper titled, “The Burden of Rare Diseases: An Economic Evaluation,” based on the results of a study which demonstrate that rare diseases impose substantial economic burden...
