A new international study has shown that treating babies with spinal muscular atrophy before symptoms appear can dramatically improve outcomes.Emily ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...

Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe and ...

Carlsbad’s Ionis Pharmaceuticals, a biotechnology company, has developed many drugs approved for the treatment of neurological disorders ...

Nathanael was 11 months old when he was diagnosed - now his family hopes screening will spare others the same wait.

Milford High School announced the hiring of Eric Western as the interim head coach of its girls wrestling program.

The complete response letter was issued by the FDA during a routine inspection of a third-party fill-finish facility, not because of an issue with apitegromab.

Biogen is working with regulatory authorities around the world to advance the high dose regimen as an additional dosing option for people living with SMA. The high dose regimen of SPINRAZA (nusinersen ...

The Kerala model is crucial as SMA patients in India had a long wait for a viable treatment option. Yet another Kerala model is now being scripted in healthcare in the treatment of rare diseases, ...

Ionis recently marked a decade of Surf Away+, a beach day of adaptive sports for people with neurological diseases, including ...