Blood Disorders | CHOP Research Institute

Blood Disorders

Published on
May 29, 2024
Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.
Published on
Jan 3, 2024
After decades of limited progress in treating sickle cell disease, we have reached an historical moment with two new gene therapies.
Published on
Oct 10, 2023
CHOP's NoT Bleeding Program will conduct clinical trials to develop the next generation of therapeutics for hemophilia A, B, and other bleeding disorders.
Published on
Jun 10, 2022
This week’s news roundup features discoveries related to COVID-19’s effect on adolescents, autism screening, telemedicine, and more.
Published on
Aug 20, 2021
This week in the news, we share findings on Fontan circulation side effects, pediatric brain tumor survivorship, gene therapy, and more.

The Dova study is for children below 18 years with primary Immune Thrombocytopenia (ITP) who have had an insufficient response to previous treatment.

Individuals with beta thalassemia that is treated with regular transfusions, and who are between the ages of 18 and 40 years old, may be able to participate.

ICON 3 is a prospective, open label, randomized, multi-center phase 3 clinical trial comparing eltrombopag with the enrolling physician’s choice of front line therapy.

Pioneering new approaches to diagnosis, care, and treatment for patients with red blood cell disorders with novel gene therapies and stem-cell-based treatments that aim to reduce symptoms and prolong life.

Published on
Nov 26, 2014
An international team of gene experts has identified a mutation that causes aplastic anemia, a serious blood disorder in which the bone marrow fails to produce normal amounts of blood cells.