More than a half million dollars was raised at the Schreiber Center for Pediatric Development’s 41st Annual Gala.

Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G) Fabhalta is the only oral alternative complement pathway ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

A mom has opened up about her daughter developing 'completely normally until one day she woke up and something was different' ...

We recently shared a list of 10 Best Short Squeeze Stocks To Buy According to Analysts. In this article, we will see how ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Neuren Pharmaceuticals (ASX:NEU) is focused on neurodevelopmental disorders and rare disease drug development.

Learn more about whether ACADIA Pharmaceuticals Inc. or Merus N.V. is a better investment based on AAII's A+ Investor grades, ...

The International Rett Syndrome Foundation (IRSF), headquartered in Cincinnati, has announced the allocation of nearly $2 million in grants to seven leading researchers, ...

Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc.

These will be to look for a genetic cause or a metabolic cause of the syndrome. Treatment for Lennox-Gastaut syndrome can be difficult, as many of the epilepsy medicines used for epilepsy seizures don ...