The US FDA announced today that it has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia. The former is notably the first FDA ...

Experts argue for new mechanisms to fund research in oft-ignored rare diseases. Newly developed gene therapies for rare diseases such as sickle cell disease (SCD) represent the pinnacle of ...

SPRING LAKE, N.C. (WTVD) -- The family of a Spring Lake 9-year-old said their daughter has shown improvements following her participation in a clinical trial for sickle cell disease gene therapy.

Recently approved gene therapies for sickle cell disease are an exciting new treatment option, but their price tags mean they are out of reach for health-care systems and individuals in lower- and ...

Andrew Campbell, MD, is a distinguished leader in the field of sickle cell disease research and treatment. Currently serving as the director of the Comprehensive Sickle Cell Disease (SCD ...

Of the 120 million people worldwide living with sickle cell, more than 66% are in Africa. Despite the prevalence, treatment to relieve pain and prevent complications is difficult to secure ...

The Somerville biotech has been able to rapidly activate qualified treatment centers for its gene therapy, but some analysts believe the number of patients starting the treatment is lagging.

Gene therapy, however, holds the promise of a transformative approach to treating SCD, potentially providing a one-time cure by addressing the root cause of the disease at the genetic level.

Bluebird Bio is making progress on the launch of its three approved gene therapies but appears to ... to their respective treatments for sickle cell disease (SCD). While Bluebird has collected ...

Purpose: The available treatment options for priapism in pediatric (1-21 years of age) patients with sickle cell disease (SCD) are reviewed. Summary: Priapism is a complication of SCD that ...