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NICE recommends Sanofi's Gaucher treatment
Gaucher is a genetic disease where glucocerebroside ... with Afinitor recommended for those with NETs of gastrointestinal or lung origin where their disease has progressed. Bain Capital has ...
Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease
FDA aligned on single-arm Phase 3 study to support potential accelerated and full approvalOn track to dose first patient in Phase 3 trial in ...
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Spur Therapeutics, FDA Align on Phase III Plans for Gaucher Disease Gene Therapy
The FDA has offered positive feedback on possible paths forward for accelerated or traditional approval of FLT201 using data from a single-arm study.
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Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease
Lyso-Gb1 is one of the best predictors of clinical response in Gaucher disease, and FLT201 showed rapid and sustained reductions in lyso-Gb1 in its Phase 1/2 GALILEO-1 trial. Full approval would ...