Recent data published in NEJM demonstrates that CRISPR gene-editing therapy achieves complete clinical efficacy endpoints in ...

Smiles from Heaven is inviting students and community members to volunteer at upcoming events while supporting local children ...

July 1 (Reuters) - The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals' gene therapy in ...

Despite missing the primary endpoint in a pediatric study, Agios says it remains steadfast in working to grow the reach of its pyruvate kinase (PK) activation franchise. Agios hopes to fill the ...

WASHINGTON — The Food and Drug Administration has approved the first drug treatment for pediatric patients 12 and older with a rare blood disorder. The treatment for acquired thrombotic ...

The era of genetic medicines has ushered in novel and exciting ways of treating genetic diseases, one of which includes bringing to reality the promise of a one-time treatment by addressing the root ...

Camp CoAg gives young patients a chance to experience summer camp in a medically safe setting ...

Immune system disorders in children can involve an underactive, overactive, or misdirected immune response. Main groups include immunodeficiencies, autoimmune diseases, atopic diseases, and ...

Children's Hospital of Philadelphia (CHOP) is proud to announce the selection of Stella T. Chou, MD, as its new Chief of the Division of Hematology. Dr. Chou served as interim chief since August 2025 ...