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Children’s Hospital of Philadelphia’s First FDA Approved Gene Therapy Patient for Severe Beta Thalassemia Celebrates Two Year Treatment Anniversary
Patient no longer needs regular blood transfusions for her inherited blood disorder, and her future has been transformed.
CHOP Named Qualified Treatment Center for Lenmeldy, the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy
CHOP’s Leukodystrophy Center, one of the leading centers of its kind in the nation, now offers Lenmeldy to treat children with metachromatic leukodystrophy (MLD).
First Patients at CHOP Begin Newly Approved Sickle Cell Gene Therapies
CHOP announced that its first patients outside of clinical trials have begun the treatment process for the newly FDA approved sickle cell gene therapies.
Researchers Publish Final Results of Key Clinical Trial for Gene Therapy for Sickle Cell Disease
In a landmark study, an international consortium led by researchers at CHOP published the final results of a key clinical trial of the gene therapy CASGEVY for the treatment of sickle cell disease.
CHOP Treats 500th CAR T Patient
CHOP has treated its 500th patient with CAR T-cell immunotherapy, a “living drug” that harnesses the power of a patient’s own immune system to fight cancer.
CHOP Will Be Provider in Independence Blue Cross’s Gene Therapy Network
CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.
CHOP Researchers Develop Tool that Reduces Errors in Stem Cell Transplant Reporting
CHOP researchers have developed a custom-built application to automate determination of engraftment, a key outcome after hematopoietic stem cell transplant.
Two CHOP Researchers Named 2022 STAT Wunderkinds
Two researchers from Children’s Hospital of Philadelphia (CHOP) were recently selected by the health and science news outlet STAT as 2022 Wunderkinds, an award that celebrates the next generation of scientific superstars who are making significant contributions to biomedical research.
CHOP Study Finds CAR T-Cell Therapy Improves Survival Regardless of Socioeconomic Status
Researchers from Children’s Hospital of Philadelphia (CHOP) have found that economically disadvantaged children treated with CAR T-cell therapy for relapsed/refractory acute lymphoblastic leukemia (ALL) fair just as well as children from more socioeconomically advantaged backgrounds.
FDA Approves First Potentially Curative Gene Therapy for Beta Thalassemia
A leading study site for nearly a decade, CHOP will be part of a network of treatment centers in the country offering the therapy to patients who require regular blood transfusions.