FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…

Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more

~Estimated

~$10 – $1,125

xCuresNCT06539169

Phase 1

An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…

Sickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Beta-Thalassemia+1 more

~Estimated

~$10 – $7,500

Daniel BauerNCT06647979

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