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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.
Children’s Hospital of Philadelphia, Penn Medicine Researchers Report High-Throughput Variant Re-Classification for Alagille Syndrome Disease Gene
Findings will help provide more accurate diagnoses to families.
Q&A with Binita M. Kamath, MBBChir, MRCP, MTR
CHOP’s new Chief of the Division of Gastroenterology, Hepatology and Nutrition shares her vision for the department’s future.
The Late Dr. Kwaku Ohene-Frempong Portrait Unveiling
The life and legacy of the late Dr. Kwaku Ohene-Frempong was celebrated by family, friends and colleagues at his portrait unveiling ceremony on CHOP’s campus on June 20.
Cure Sickle Cell Walk & Family Fun Day Presented by West Pharmaceutical Services Raises More Than $256,000 for Sickle Cell Disease Research and Care
Earlier this month, Children’s Hospital of Philadelphia (CHOP) hosted the Cure Sickle Cell Walk & Family Fun Day presented by West Pharmaceutical Services at Citizens Bank Park raising more than $256,000 (and counting!) to support sickle cell warriors.
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.
FDA Approves Two Gene Therapies for Sickle Cell Disease
The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
Researchers Develop “In Vivo” Gene Editing Model for Blood Disorders
CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.
Dr. Alexis Thompson Discusses Early Successes of Gene Therapy for Sickle Cell Disease
In a short documentary by the New England Journal of Medicine, Dr. Alexis Thompson discusses the early successes of gene therapy, as well as its risks and benefits.
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.