by CheckRare Staff | Sep 12, 2023
Acromegaly, a serious and rare disease caused by a pituitary adenoma, affects thousands of individuals worldwide. The excess secretion of growth hormone (GH) leads to the production of insulin-like growth factor 1 (IGF-1), resulting in systemic complications and...
by CheckRare Staff | Sep 6, 2023
Shoshana Shendelman, PhD, President and Chief Executive Officer of Applied Therapeutics, discusses the results of the Phase 3 clinical trial testing govorestat to treat galactosemia. Galactosemia is a rare, metabolic disorder that affects a person’s...
by CheckRare Staff | Sep 5, 2023
Mona Al Mukaddam, MD, Associate Professor of Clinical Medicine and Orthopaedic Surgery at Penn Medicine, describes fibrodysplasia ossificans progressiva (FOP). FOP is genetic disorder characterized by new bone formation outside of the normal skeletal...
by CheckRare Staff | Sep 4, 2023
Shoshana Shendelman, PhD, President and Chief Executive Officer of Applied Therapeutics provides a brief overview of the orphan drug company. As Dr Shendelman explains, Applied Therapeutics is a biopharmaceutical company developing a pipeline of novel...
by CheckRare Staff | Aug 28, 2023
Mary P. McGowan, MD, chief medical officer of the Family Heart Foundation provides an overview of homozygous familial hypercholesterolemia (HoFH). HoFH is a rare genetic disorder that results in severe impairment of the low-density lipoprotein receptor...
