DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.

It had all the makings of a typical graduation party. Blue and yellow streamers lined the room. Signs were posted on the walls that said “Congrats Grad 2025,” “You did it” and “Made the grade.” In the ...

Queensburgh Bowling Club recently bought stickers to support the Reach for a Dream Foundation's Slipper Day initiative.

A 46-year-old woman with type 1 myotonic dystrophy (DM1) presented to the emergency department with dyspnea and palpitations.

With PIP reforms proposed in Parliament, how long could people feel the sting if they were denied their claim?

The U.S. FDA has paused Rocket Pharmaceuticals' mid-stage gene therapy trial after a patient died from serious complications, ...