"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: ...
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Scholar Rock In Spotlight After FDA Refuses To Approve Muscular Atrophy Treatment Over Third-Party Manufacturing Facility Issues
Shares of Scholar Rock (SRRK) rose about 2% on Tuesday morning after falling 12% in the pre-market session after the U.S.
"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 ...
Scholar Rock navigates FDA's Complete Response Letter for Apitegromab, aiming for swift resubmission to address spinal ...
FDA cites Catalent Indiana issues in Scholar Rock's SMA drug review; European approval decision expected in 2026, with ...
SMA type 3 is a hereditary disease that affects motor neurons. People may also refer to it as Kugelberg-Welander disease. Symptoms usually begin after 18 months of age. Children with SMA type 3 can ...
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