Gene therapy treatment gives Baby Kiama a lifeline, saving him from world’s most expensive drug that he needed to slow down a ...

A majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe ...

“We were referred to a paediatrician who put him on reflux medication. However, things were getting worse,” Ellie said. “He ...

Chugai Pharma’s Evrysdi tablets 5mg receives Japanese approval to treat spinal muscular atrophy: Saturday, March 29, 2025, 11:00 Hrs [IST] Chugai Pharmaceutical Co., Ltd. announ ...

Lumenis announced the launch of OptiLift, a dynamic muscle stimulation technology intended to improve lower eyelid laxity, ...

PTC Therapeutics said on Friday the European Commission will remove its inherited progressive muscle-wasting disorder treatment from the region after an advisory panel reaffirmed a negative opinion of ...

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

Scholar Rock (SRRK) announced that the U.S. Food and Drug Administration FDA has accepted its Biologics License Application BLA for ...

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...