HYDROXYUREA INTOLERANCE

Clinical trials for HYDROXYUREA INTOLERANCE explained in plain language.

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CRISPR breakthrough: One-Shot gene therapy aims to free kids from sickle cell pain crises

Disease control Ongoing

This study is testing a one-time treatment called CTX001 in children with severe sickle cell disease who don't respond well to the standard medication, hydroxyurea. The treatment uses the patient's own modified stem cells (a type of gene therapy using CRISPR technology) to try to…

Matched conditions: HYDROXYUREA INTOLERANCE

Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control

Last updated Mar 20, 2026 14:48 UTC

HYDROXYUREA INTOLERANCE

Get alerted when new HYDROXYUREA INTOLERANCE trials appear

CRISPR breakthrough: One-Shot gene therapy aims to free kids from sickle cell pain crises