Randomized Trialhigh evidence
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
The New England journal of medicine·May 9, 2024·PMID 38661449
Why it mattersExa-cel offers a potentially curative, one-time intervention that nearly eliminates vaso-occlusive crises—the primary driver of morbidity and healthcare utilization in sickle cell disease—using a nonviral gene-editing approach.
The papers that matter, first.
MedLit ranks the medical literature into a specialty feed with concise summaries, evidence-aware triage, and visual takeaways.
§
MedLit · Evidence, Distilled