Ascendis’ likely approval of TransCon CNP for achondroplasia poses a major threat to BioMarin’s best-selling drug. Read why I ...

Evan Winter was diagnosed him with Duchenne muscular dystrophy (DMD) earlier this year which only around 2,500 people have in ...

Roche has halted some international shipments of the gene therapy Elevidys, used to treat Duchenne muscular dystrophy, Bloomberg reported July 22. The decision comes in the wake of a similar move by ...

A timeline of key developments surrounding Sarepta Therapeutics' Duchenne muscular dystrophy drug Elevidys, and the potential ...

For Sarepta, the hits keep coming, with several downgrades in the past week after it was forced to temporarily take its ...

The Food and Drug Administration won’t sign off on Sarepta Therapeutics Inc. bringing its controversial gene therapy back to ...

Little Evan, who lives in Maidstone in Kent, was only told he had the debilitating condition on February 8 this year after ...

Bank of America downgraded Sarepta Therapeutics (NASDAQ:SRPT) to Underperform from Neutral on Wednesday, becoming the latest brokerage to issue a bearish view on the company, citing safety concerns ...

The Swiss company, which markets Elevidys abroad, said it believes the benefit-risk balance to treatment remains positive in ...

In this week’s edition of InnovationRx, we look at Moderna’s use of quantum computers, a new top drug regulator at the FDA, ...

Roche is pausing shipments of Elevidys in some countries, following partner Sarepta Therapeutics’ move in the U.S., as safety ...

A public workshop that columnist Shalom Lim and his girlfriend, Amanda, organized aimed to foster connection among people ...