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.
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.
The ex vivo gene therapy improved sulfatase production and reduced symptoms associated with the disease in preclinical models
Researchers at CHOP announced the results of a new study offering insights into the development of next-generation gene therapies to treat hemophilia A.
Researchers at Children’s Hospital of Philadelphia (CHOP) revealed for the first time that children, adolescents and young adults may experience very rare neurological issues of paraparesis and quadriparesis following chimeric antigen receptor T-cell (CAR-T) therapy, a type of immunotherapy used to treat B-cell Acute Lymphoblastic Leukemia (B-ALL). Additionally, these complications occurred without the type of inflammation typically seen in adults. The findings were published today in the journal Blood.
Researchers in the Division of Orthopedics and Sports Medicine and Performance Center at Children’s Hospital of Philadelphia (CHOP) announced results from a large retrospective study of patients surgically treated for Patellar Sleeve Fractures (PSFs), rare knee fractures that occur when a piece of bone and cartilage separates from kneecap. They found that in most pediatric patients with displaced fractures, surgery was effective in restoring full range of motion (ROM) and enabled them to return to their regular activities.
CHOP’s new Chief of the Division of Gastroenterology, Hepatology and Nutrition shares her vision for the department’s future.
Findings provide some of the first evidence of potential therapeutic targets for condition affecting thousands of patients with single ventricle congenital heart disease.
By helping researchers cluster and analyze new data, new drug targets for rare diseases could be identified.
New method, bolstered by a study of 89 million points of data, could help patients receive precision care months or years earlier