The Food and Drug Administration’s (FDA’s) Division of Neurology has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019.
Luxturna: Gene Therapy for Biallelic RPE65 Mutation-Associated Retinal Dystrophy
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company’s first approved product, Luxturna — a one-time gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
Sickle Cell Disease: Overview and Emerging Stem Cell Transplant Therapy
Mitchell Cairo, MD, Chief of Pediatric Hematology, Oncology and Stem Cell Transplantation at New York Medical College discusses his study at the 2018 American Society of Hematology (ASH) Annual Meeting in San Diego focusing on Sickle cell disease, a group of inherited red blood cells disorders.
Morquio and Maroteaux-Lamy Syndrome (MPS IV and VI) Overview
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City provides an overview of Mucopolysaccharidosis type IV and VI (MPS IV and VI). Mucopolysaccharidosis type IV is also known as Morquio syndrome, is a progressive condition that mainly affects the skeleton.
Lysosomal Storage Disorders
Lysosomal storage disorders (LSDs) are a group of about 50 genetic diseases caused by defects in lysosomal proteins or lysosomal related-proteins, which results in dysfunction of lysosomes.
FDA Grants Priority Review for Edsivo NDA for the Treatment of vEDS
The U.S. Food and Drug Administration (FDA) has accepted for review a new drug application (NDA) for Edsivo for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation.
