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Scholar Rock Receives CRL from FDA for Spinal Muscular Atrophy Treatment Apitegromab
23 Sep 2025 19:19 GMT
… treatment of patients with spinal muscular atrophy (SMA). According to Scholar Rock … Treatment for Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations …
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Scholar Rock In Spotlight After FDA Refuses To Approve Muscular Atrophy Treatment Over Third-Party Manufacturing Facility Issues
23 Sep 2025 18:13 GMT
… treatment of patients with spinal muscular atrophy (SMA).
The company stated that … function in patients with Spinal Muscular Atrophy (SMA), a rare, severe, genetic …
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Scholar Rock's Spinal Muscular Atrophy Drug Faces FDA Delay Over Catalent Indiana Inspection
23 Sep 2025 15:56 GMT
… License Application (BLA) for spinal muscular atrophy (SMA).
The CRL is related …
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Much Maligned Manufacturing Site Trips Up Scholar Rock’s Spinal Muscular Atrophy Drug
23 Sep 2025 12:59 GMT
… myostatin blocker apitegromab for spinal muscular atrophy, citing issues at a third …
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FDA hands Scholar Rock a CRL for spinal muscular atrophy drug
23 Sep 2025 12:19 GMT
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FDA Issues Complete Response Letter (CRL) for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations Identified at Catalent Indiana LLC Fill-Finish Facility
23 Sep 2025 11:53 GMT
… children and adults with spinal muscular atrophy (SMA) and additional rare, severe … -targeted treatment candidate in spinal muscular atrophy (SMA) to demonstrate clinical success … children and adults with spinal muscular atrophy (SMA) and other rare, severe …
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Clinical trial reveals encouraging results for apitegromab in spinal muscular atrophy
18 Sep 2025 19:23 GMT
… children and adolescents with spinal muscular atrophy, according to a study published … in The Lancet Neurology.
Spinal muscular atrophy (SMA) is a rare genetic … 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double …
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US FDA declines to approve higher dose of Biogen's genetic disorder drug
23 Sep 2025 20:45 GMT
… in 2016 to treat spinal muscular atrophy, which is the leading inherited … efforts to revitalize its spinal muscular atrophy franchise, which has been losing …
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Biogen Provides Regulatory Update on High Dose Regimen of Nusinersen
23 Sep 2025 20:30 GMT
… for the treatment of spinal muscular atrophy (SMA). The FDA letter requested … , children and adults with spinal muscular atrophy (SMA). As a foundation of …
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FDA Delays Apitegromab Approval, Cites Facility Concerns
23 Sep 2025 21:11 GMT
… treatment of patients with spinal muscular atrophy (SMA), according to a news … muscular component of SMA.”
Spinal muscular atrophy is a rare, progressive and …
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