Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Apa yang diuji dalam uji klinis ini
This is the study that made Casgevy an approved medicine. Participants with severe sickle cell disease had their own blood stem cells edited and returned. The main question was whether they would stop having the severe pain crises that define the disease — and for the large majority, they did.
An open-label, single-arm, multi-site Phase 2/3 study of autologous CRISPR-Cas9-modified CD34+ cells (CTX001, later exagamglogene autotemcel) in patients aged 12–35 with severe sickle cell disease. The primary endpoint was freedom from severe vaso-occlusive crises for at least 12 consecutive months. Results supported approvals by the MHRA, FDA and EMA.
Sources
- New England Journal of Medicine · 2024
Exagamglogene autotemcel for severe sickle cell disease ↗
Apa yang dimaksud dan tidak dimaksud oleh hasil uji klinis
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