News

People with sickle cell disease (SCD) in Ghana — a country in sub-Saharan Africa, a region with the world’s highest SCD prevalence — often go undiagnosed until age 4 and during a pain crisis, a study into patients in Accra, the country’s capital, reported. These findings reflect the importance…

A new treatment, called Adakveo (crizanlizumab), is now available to treat sickle cell disease (SCD) patients — the first time in decades that a new therapy for the disease has been made accessible — through the United Kingdom’s National Health Service (NHS), according to the U.K.

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

Two years since its establishment, the Networking California for Sickle Cell Care initiative has seen increasing numbers of specialized clinics and healthcare professionals knowledgeable about sickle cell disease (SCD) and is setting new goals to improve the lives of patients in the state, the Network announced in its…

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…