Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...

The main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was hobbled by manufacturing concerns at a Novo Nordisk facility last year but ...

But for Brooklyn, who lives with spinal muscular atrophy, or SMA, a rare genetic condition that affects muscle strength and ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

Parents have said new screening tests for Spinal muscular atrophy are a "game-changer".

A crowdfunding campaign for five-month-old Ginny, a Singaporean baby diagnosed with Spinal Muscular Atrophy (SMA) Type 1, ...

Ashley Webb, MD, Program Director, Pediatric Neurology, at the University of Texas Health Science Center at Houston McGovern Medical School, discusses the need to make genetic therapy more accessible ...

All parents will now be offered spinal muscular atrophy screening for their newborns.

After a year of waiting, the Ministry of Public Health and the High Cost Drugs Program have yet to approve the life-saving medicine for baby Dayron. “Without the medicine, Dayron Almonte will not be ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...