News

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…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Several clinical research sites seeking to find new treatments for people with sickle cell disease (SCD) are joining a collaborative network created by the American Society of Hematology Research Collaborative (ASH RC). The initiative, called ASH RC Sickle Cell Disease Clinical Trials Network, has already engaged about 24,000 patients…

Since Sept. 21, 1983, when then-President Ronald Reagan’s proclamation created National Sickle Cell Anemia Awareness Month, advocates, patients, family members, doctors, and others impacted by sickle cell disease (SCD) have been pushing for increased research and awareness, and better treatments. This year will be no different, as virtual,…

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…