Despite being the only person diagnosed with all three subtypes of pyoderma gangrenosum, Becker remains hopeful. "There’s no ...

Anthony Monaco was a very active teen—involved in soccer, wrestling, and even cross country—but he always felt a little awkward physically. It started with running a little differently than the kids ...

Rutgers faculty elected to the newest class of fellows of the American Association for the Advancement of Science (AAAS) are ...

With drugs on the horizon for treating and possibly preventing transthyretin amyloid cardiomyopathy, cardiologists are ...

Shares of Soleno Therapeutics jumped 40% in morning trading on Thursday, after the company's drug for a rare metabolic condition became the first such treatment to receive U.S. regulatory nod.

Ionis Pharmaceuticals IONS signed a license agreement with Sweden-based Sobi. Per the terms, Sobi will acquire exclusive ...

Vykat XR is specifically indicated to address hyperphagia, or the abnormally strong sensation of hunger, which often leads to ...

An Enniskillen mother fears that her son – who suffers from a rare genetic condition – will be one of thousands to lose out amid ...

The legislation would provide training and compensation for parents and likewise caregivers who provide at home medical care for medically fragile children. While the bill received support from NY ...

Patients with Fuchs endothelial corneal dystrophy with particular genetic markers are at higher risk for surgery and vision loss.

Researchers studying over 2,000 Ashkenazi Jewish centenarians uncovered two rare IGF-1 gene variants linked to exceptional ...

Cerebrocostomandibular syndrome is a rare genetic disorder characterized by a combination of abnormalities affecting the ...