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CHOP Researchers Develop Innovative Model Offering New Insights to Improve and Overcome Current Obstacles in Hemophilia A Gene Therapy

Sep 5, 2024

Researchers at CHOP announced the results of a new study offering insights into the development of next-generation gene therapies to treat hemophilia A.

Children’s Hospital of Philadelphia Researchers Reveal New Hope for Chemotherapy-Resistant Neuroblastoma Patients

Aug 13, 2024

Researchers at Children’s Hospital of Philadelphia (CHOP) announced a significant breakthrough in understanding chemotherapy resistance in high-risk neuroblastoma, a common and potentially deadly childhood cancer arising within the peripheral nervous system.

The Late Dr. Kwaku Ohene-Frempong Portrait Unveiling

Jul 1, 2024

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.

Researchers at Children’s Hospital of Philadelphia Find Lentiviral Gene Transfer Improves Human Alpha Globin Production for the Treatment of Alpha Thalassemia

Jul 1, 2024

Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a new model that offers a potential platform for developing novel therapies to treat Alpha Thalassemia (AT), a severe blood disorder. The findings were published in the journal Blood.

Cure Sickle Cell Walk & Family Fun Day Presented by West Pharmaceutical Services Raises More Than $256,000 for Sickle Cell Disease Research and Care

Jun 20, 2024

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.

CHOP Researchers Develop Safer, Alternative Conditioning Regimen for the Treatment of Beta-Thalassemia

May 16, 2024

Innovative model highlights bone marrow transplantation without toxic side effects.

New Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment

May 13, 2024

New model and vector may hold the keys to transforming the lives of XLSA patients.

Researchers Publish Final Results of Key Clinical Trial for Gene Therapy for Sickle Cell Disease

Apr 24, 2024

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.

COVID-19 Vaccine Reduces Long COVID in Children

Jan 16, 2024

Vaccination associated with moderate protection in large, diverse cohort.

FDA Approves Two Gene Therapies for Sickle Cell Disease

Dec 8, 2023

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).

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