The biotech is now banking on secondary data signals as it progresses ignaseclant to late-stage trials in Charcot-Marie-Tooth ...

A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...

Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and ...

Novartis could have gone anywhere to develop lifesaving drugs. It chose to bet big on San Diego scientists. The Swiss pharma giant broke ground on its $1.1 billion research campus in Sorrento Mesa on ...

9-1-1: Nashville star Isabelle Tate’s death is bringing attention to a rare disease. On Thursday, October 23, news broke that the actress had died at the age of 23. Her agency confirmed the news via ...

Actress Isabelle Tate, who appeared as Julie in ABC’s 9-1-1: Nashville, tragically passed away at the age of 23 on October 19, 2025. Tate had a rare form of Charcot-Marie-Tooth disease (CMT), a ...

(Reuters) - Using technology gleaned from the mapping of the human genome, privately held French drug developer Pharnext SAS is a step closer to providing a treatment for a rare neurological disorder ...