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

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 ...

"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 ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

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

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 ...

Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and ...

Despite possessing a solid data package on the potential of high-dose Sprinraza in spinal muscular atrophy (SMA), Biogen will ...

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

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

The denial sets back a drug Scholar Rock hopes to become part of a new standard of care for spinal muscular atrophy, treatment for which has changed dramatically over the last decade.