Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE clinical trial - - Expect to complete enrollment in ACHIEVE ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

Myotonic dystrophy type 1 incurs high healthcare costs, driven by age and comorbidities like cardiac and respiratory issues. Early detection and integrated management of multisystemic complications ...

The comedian, who died Tuesday at 67, was dealing with "a very specific, rare genetic disorder," cardiologist Dr. Jennifer Haythe tells PEOPLE Julie Mazziotta is the Senior Sports Editor at PEOPLE, ...

Comedian Gilbert Gottfried, shown in a 2016 photo, died on Tuesday, April 12, from recurrent ventricular tachycardia due to myotonic dystrophy type II. Scott Roth Scott Roth/Invision/AP Comedian ...

PHOENIX - We are learning more about the death of comedian Gilbert Gottfried, which was announced on April 12. According to a statement from Gottfried's longtime friend and publicist, Glenn Schwartz, ...

A PepGen drug in development for myotonic dystrophy type 1 (DM1) has encouraging early human data that suggest it could be a better treatment than other medicines in clinical testing for this rare, ...

Caitlin O'Kane is a New York City journalist who works on the CBS News social media team as a senior manager of content and production. She writes about a variety of topics and produces "The Uplift," ...

On Tuesday, Gilbert Gottfried's family shared the sad news that the comedian had died at age 67 after a "long illness." Gottfried, who famously voiced Iago in Disney's 1992 classic Aladdin, had been ...