by Peter Ciszewski | Jan 8, 2021
Judy Shizuru, MD, Blood and Marrow Transplant Specialist, Stanford University School of Medicine, gives an overview of severe combined immune deficiency (SCID). As Dr. Shizuru explains, SCID is a group of inherited immune system disorders characterized by...
by Peter Ciszewski | Jan 8, 2021
Elizabeth Thompson, PhD at Horizon Pharmaceuticals provides an overview of thyroid eye disease (TED), a rare autoimmune disease causing permanent facial disfigurement severely affecting patients’ quality of life and daily function. The disease often...
by Peter Ciszewski | Jan 8, 2021
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics at Cincinnati Children’s Hospital Medical Center, gives an overview of Fabry disease. As Dr. Hopkin explains, Fabry disease is a rare X-linked lysosomal storage disorder that results in the cellular...
by Peter Ciszewski | Jan 7, 2021
Professor Ulla Feldt-Rasmussen, MD, Department of Medical Endocrinology and Metabolism, Rigshospitalet Copenhagen University Hospital, gives an overview of hypothalamic obesity (HO). HO is a rare endocrine disorder characterized by severe and debilitating...
by Peter Ciszewski | Jan 7, 2021
Michael Hayden, PhD, Chief Executive Officer of Prilenia Therapeutics, talks about the PROOF-HD trial currently underway to treat Huntington disease. Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting...
