Medicine
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NEJM published first pediatric data for exa-cel in children under 12 with sickle cell disease or transfusion-dependent beta thalassemia.
Why now
CRISPR medicine is moving younger, raising the early-intervention question before years of organ damage accumulate.
BridgeBio’s once-daily oral infigratinib improved annualized height velocity and body proportionality in a Phase 3 achondroplasia trial published in NEJM.
Why now
Oral therapy could change the treatment-burden conversation for achondroplasia if regulators agree.
Penn's decade-long follow-up gives CAR-T cure language a stronger footing for a subset of lymphoma patients, while showing why not everyone should be promised the same outcome.
Why now
A 10-year follow-up gives CAR-T storytelling the missing timeline: not just response, but durable remission in some of the earliest lymphoma patients treated.
Penn researchers used dual CAR-T therapy to reduce harmful antibodies in two highly sensitized kidney candidates, enabling both to receive kidney transplants.
Why now
A cancer-born cell therapy may have opened a transplant path for patients who were almost impossible to match.
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