Urgency For Galactosemia Treatment

Shoshana Shendelman, PhD, President and Chief Executive Officer of Applied Therapeutics, explains the urgency of finding newer treatment options for galactosemia patients.     Galactosemia is a rare, metabolic disorder that affects an individual’s...

Lipid Profiles in Children

Mary McGowan, MD, chief medical officer of the Family Heart Foundation explains when it is recommended that children be screened with a lipid profile.    T   The American Academy of Pediatrics and the National Heart, Lung, and Blood Institute of the NIH...

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...