by James Radke, PhD | Nov 21, 2022
Mariana C. Castells MD, PhD, Division of Allergy and Clinical Immunology, Brigham and Women’s Hospital, and Professor of Medicine, Harvard Medical School, gives an overview of mastocytosis. As Dr. Castells explains, mastocytosis is a rare, hematologic...
by James Radke, PhD | Oct 18, 2022
Jennifer McNary, co-founder of One Rare, discusses this innovative new program designed to improve the experiences of young adults with rare diseases. As Ms. McNary explains, a lot of patient advocacy support is aimed toward young children. However, as more...
by James Radke, PhD | Sep 30, 2022
The US Food and Drug Administration (FDA) has approved the combination oral medication of sodium phenylbutyrate and taurursodiol (Relyvrio) to treat individuals with amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease, characterized by the...
by James Radke, PhD | Sep 29, 2022
The U.S. Food and Drug Administration (FDA) has approved dupilumab to treat adults with prurigo nodularis. Prurigo nodularis is an inflammatory skin disorder characterized by extreme itching and symmetrical rashes. Most areas commonly affected are the arms, legs, the...
by James Radke, PhD | Sep 19, 2022
The U.S. Food and Drug Administration (FDA) has granted accelerated approval for elivaldogene autotemcel (el-cel) to treat males 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). CALD is a progressive neurodegenerative disease that involves...
