by Peter Ciszewski | Jun 6, 2022
Gregory Davis, PhD, Vice President of Genome Engineering at Sangamo Therapeutics, explains why the use of genomic medicines, such as Sangamo’s proprietary zinc finger protein technology, are complementary to many rare diseases. Zinc finger proteins are...
by Peter Ciszewski | Jun 2, 2022
Tomek Szczudlo, VP, Global Medical Affairs, Franchise Head, Solid Tumors at Daiichi Sankyo, lists diagnostic delays as a key priority that physicians should be aware of about tenosynovial giant cell tumors (TGCT). TGCT are a group of rare, benign tumors that...
by Peter Ciszewski | Jun 1, 2022
Barbara Burton, MD, Attending Physician, Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children’s Hospital of Chicago, gives an overview of alpha mannosidosis. As Dr. Burton explains, alpha mannosidosis is a multisystemic,...
by Peter Ciszewski | May 31, 2022
Deirdre Kelly, MD, FRCP, FRCPI, FRCPCH, Consultant Pediatric Hepatologist, Liver Unit, Birmingham Women’s and Children’s Hospital and University of Birmingham, the National Institute for Health and Care Excellence (NICE) officially announcing its recommendation of...
by Peter Ciszewski | May 30, 2022
Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, provides an extensive overview of mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome. In...
