by Peter Ciszewski | May 22, 2019
The US Food and Drug Administration (FDA) has cleared Abeona Therapeutics to begin a Phase 1/2 clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease. ABO-202 is designed to deliver a functional copy of the PPT1 gene to...
by Peter Ciszewski | May 22, 2019
The US Food and Drug Administration (FDA) has extended the indication for teduglutide (Gattex, Takeda) injection to children as young as 1 year old with short bowel syndrome (SBS) who need additional intravenous parenteral support. Teduglutide is the only...
by Peter Ciszewski | May 22, 2019
Jeffrey W Olin, DO, Professor of Medicine at the Icahn School of Medicine at Mount Sinai, discusses the signs and symptoms of fibromuscular dysplasia (FMD) and recent research in this rare condition. FMD is characterized by abnormal development or growth of...
by Peter Ciszewski | May 20, 2019
Debra Miller is the founder and CEO of CureDuchenne, and in this video she discusses some of the current clinical research in Duchenne muscular dystrophy (DMD) and the hope of a possible cure. Debra founded CureDuchenne in 2003 with her husband after their only...
by Peter Ciszewski | May 17, 2019
Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the common early features of mucopolysaccharidoses (MPSs), a...
