News

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

The National Institutes of Health (NIH) has awarded a $10.5-million grant to support several research projects seeking to improve care for people with sickle cell disease (SCD). Research will focus on children and young adults, and on combating racism and other barriers to proper healthcare. SCD disproportionately affects Black…

Seeking to improve the health and life quality of underserved sickle cell disease (SCD) communities internationally, Global Blood Therapeutics (GBT) has established The GBT Foundation. Created in part to mark the company’s 10 anniversary, the foundation will fund programs around the world that support SCD patients, their families,…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias. Ferriprox’s use was also approved in combination with other iron-binding, or iron-chelating, treatments for people with thalassemia major, a severe…

The 10th Annual Sickle Cell Disease Therapeutics Conference, to be held virtually Sept. 15, will highlight the most recent medical advances and developing treatment trends in the field of sickle cell disease (SCD). Taking place during National Sickle Cell Awareness Month, the conference will be hosted by the…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

The U.S. Food and Drug Administration (FDA) has agreed to consider Global Blood Therapeutics’ (GBT) request to expand the use of Oxbryta (voxelotor) to children as young as 4 with sickle cell disease (SCD). The oral therapy is currently conditionally approved — when given at a daily…

FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, was found to be effective at raising the levels of fetal hemoglobin in healthy adults enrolled in a Phase 1 trial, according to new interim results. Study data also indicated that FTX-6058 was…