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

Leslie Raffini, MD, and Regina Butler, RN, were quoted in the Philadelphia Inquirer on a story featuring a CHOP patient family living with hemophilia, an inherited bleeding disorder that prevents blood from clotting properly.

A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.

CHOP gene therapy expert Dr. Lindsey George reacts to a phase 3 hemophilia gene therapy study in an editorial in the New England Journal of Medicine.

CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.

Two new treatments are giving children with spinal muscular atrophy hope. CHOP is on the forefront of delivering care that is changing children’s lives.

Find out how a bold idea led to an astonishing cure for a certain childhood blindness, and learn about the future of gene therapy to treat children, like Hannah.

Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.

A breakthrough therapy has its genesis in more than 10 years of research conducted at Children's Hospital of Philadelphia.