by Peter Ciszewski | Jan 13, 2021
Michael Hayden, PhD, Chief Executive Officer of Prilenia Therapeutics, describes the disheartening impact a diagnosis of Huntington disease can have on a family. Huntington disease is an inherited, neurodegenerative disorder. If a parent has the condition,...
by Peter Ciszewski | Jan 12, 2021
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics at Cincinnati Children’s Hospital Medical Center, discusses how clinicians and patients with Fabry disease choose the best treatment option. Fabry disease is a rare X-linked lysosomal storage...
by Peter Ciszewski | Jan 11, 2021
Judy Shizuru, MD, Blood and Marrow Transplant Specialist, Stanford University School of Medicine, discusses clinical data from an ongoing phase 1 trial of JSP191 in patients with severe combined immune deficiency (SCID). SCID is a group of inherited immune system...
by Peter Ciszewski | Jan 11, 2021
Ulla Feldt-Rasmussen, MD, Department of Medical Endocrinology and Metabolism, Rigshospitalet Copenhagen University Hospital, discusses results from a phase 2 open-label extension study of tesomet to treat patients with hypothalamic obesity (HO). HO is a rare...
by Peter Ciszewski | Jan 10, 2021
Lisa Nachtigall, MD, Clinical Director, Neuroendocrine and Pituitary Clinical Center at Massachusetts General Hospital, and Associate Professor at Harvard Medical School discusses acromegaly and pituitary tumors. Acromegaly is a rare disorder caused by a...
