A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia

Fulcrum scraps sickle cell drug program after FDA raises cancer-risk concerns

Fulcrum scraps sickle cell drug program after FDA raises cancer-risk concerns

MATCH cohort provides regulatory-ready external control framework for MSUD gene therapy trials

This article presents the MSUD Age-matched Standard Treatment Cohort (MATCH), a prospective natural history study of 11 infants with classic

MATCH cohort provides regulatory-ready external control framework for MSUD gene therapy trials

This article presents the MSUD Age-matched Standard Treatment Cohort (MATCH), a prospective natural history study of 11 infants with classic

Fulcrum plummets over 50% after scrapping lead sickle-cell drug on FDA concerns

https://doi.org/10.1056/NEJMoa2517384

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MRI end-points for clinical trials in ataxias: recommendations from the Ataxia Global Initiative MRI Biomarkers Working Group