<![CDATA[FDA Expands Wilate Approval for Routine Prophylaxis in Children Younger Than 6 With von Willebrand Disease]]>
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Gene therapy for children with sickle cell disease approved by FDA
A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.

MATCH cohort provides regulatory-ready external control framework for MSUD gene therapy trials
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MATCH cohort provides regulatory-ready external control framework for MSUD gene therapy trials
Brigatti et al. develop MATCH, a prospective natural history cohort designed as a regulatory-ready external control for gene therapy trials

FDA Approves First Gene Therapy For Young Children With Sickle Cell Disease
The report said a clinical trial involving 15 children between the ages of 5 and 11 produced encouraging results.
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