"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 ...

When we think about genetic diseases, the ones that usually come to mind are cystic fibrosis, Fragile X and, of course, Tay-Sachs disease. One disease that many people are not aware of is spinal ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

(WNDU) - Imagine your baby being born healthy, and then within months, they start to lose the ability to move their arms and legs, fingers, and toes. This is what happens to infants born with spinal ...

On Tuesday, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Biogen Inc.’s (NASDAQ: ...

Shares of Scholar Rock (SRRK) rose about 2% on Tuesday morning after falling 12% in the pre-market session after the U.S.

Scholar Rock navigates FDA's Complete Response Letter for Apitegromab, aiming for swift resubmission to address spinal muscular atrophy treatment needs.

"The benefits of newborn screening identified in the study presented here encompass not only the achievement of motor milestones, but also a reduced need for ventilator support or tube feeding and a ...

FDA cites Catalent Indiana issues in Scholar Rock's SMA drug review; European approval decision expected in 2026, with ...

The regulatory action marks the second rejection for a spinal muscular atrophy therapy this week after Scholar Rock’s ...

In its rejection letter, the FDA flagged problems at a third-party fill-finish site owned by Novo Nordisk. Issues at this ...

RUSH CITY, Minn. — Rush City native Tristan Larsin is a very successful app developer —he works for Zillow. But his climb to success involved some extra hurdles — hurdles he's taking away for others. ...