What is Ben’s Friends

Ben Munoz, co-founder of Ben’s Friends, talks about his non-profit organization.     Ben’s Friends is a 501(c)(3) non-profit designed to provide a network for patients with rare diseases or chronic illnesses. Its goal is to be a safe and supportive place for...

A Possible Breakthrough in Acromegaly Treatment

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...

Clinical Trial Testing Govorestat

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...

FOP Explained

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...

Applied Therapeutics Overview

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...