Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and ...

Dubai: A Saudi research team from Qassim University has discovered an extremely rare genetic disorder, Bethlem Myopathy, for ...

Nashville, Tennessee Friday, November 8, 2024, 13:00 Hrs [IST] ...

Neurotech has appointed Dr Anthony Filippis as its managing director to steer the company as it progresses clinical testing ...

“We need help from other people,” pleaded Pavlina. “We saw videos from the children who were already there, it [the treatment ...

NHS data shows nearly one in five children waited more than 18 weeks for chairs or modifications between June 2023 and June ...

Columnist Betty Vertin wishes she could take away the pain over the milestones in the teenage years her sons with DMD will miss.

According to the Mayo Clinic, muscular dystrophy is a group of diseases that cause loss of muscle mass and progressive ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment ...

there are at least 21 different monogenic causes of muscular dystrophy, and cardiovascular complications are commonly associated with some subtypes. [1] In normal skeletal muscle, there is a ...

Investors consider Dyne Therapeutics' treatments for muscle diseases and strong cash position as a speculative Buy ...

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...