Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD). But the company’s efforts to expand its use to a new indication is coming ...

With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. Late Thursday, PTC revealed that it ...

Sarepta shares rise more than 12% Analysts see potential for improved perception, commercial uptake Elevidys shows up to 73% reduction in rate of functional decline Jan 26 (Reuters) - Sarepta ...

(RTTNews) - Shares of REGENXBIO Inc. (RGNX) are up over 11% at $16.27 in premarket trading on Monday, following encouraging new long-term data for its Duchenne program and key catalysts lined up for ...

The Scottish Medicines Consortium (SMC) has accepted givinostat (Duvyzat) for restricted use in NHS Scotland for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 6 years or older.

Gait apraxia is characterized by difficulty coordinating and initiating the movement of walking. It is distinct from lower limb apraxia, which may affect motor function of the lower limbs more ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

Sarepta Therapeutics (SRPT) shares fell after its late-stage trial for two gene therapies, AMONDYS 45 (casimersen) and VYONDYS 5 (golodirsen), aimed at treating Duchenne muscular dystrophy, did not ...

People with Duchenne muscular dystrophy have a shorter-than-average life expectancy due to changes in muscles that affect breathing, heart activity, and other functions. However, treatment advances ...

Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver ...

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...

People with Duchenne muscular dystrophy (DMD) frequently live well into adulthood. Advances in treatment have significantly increased the life expectancy for this condition. The median life expectancy ...