News

Emmaus Life Sciences is seeking the approval of Endari (L-glutamine), an oral treatment for sickle cell disease (SCD), in the the United Arab Emirates (UAE). “We look forward to working with the U.A.E Ministry of Health to bring this important medication to our patient population in the…

Scientists Kenneth Ataga, MD, and Santosh Saraf, MD, have received a $3.2 million grant to continue investigating the potential of machine learning as a method to help identify sickle cell disease (SCD) patients at high risk of developing progressive kidney disease. The grant awarded by the…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

GPH101, Graphite Bio’s investigational gene editing therapy, is able to correct the disease-causing mutation in blood stem cells of people with sickle cell disease (SCD), new preclinical data shows. Graphite also announced it remains on track to start enrolling participants in a Phase 1/2 trial testing the gene…

Health Canada has expanded its approval of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to patients who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The decision comes about five months after the U.S. Food and Drug Administration (FDA) approved…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

The Sickle Cell Community Consortium is gearing up for its inaugural Caregiver Summit, to be held virtually Nov. 12–14. The event is being hosted in partnership with Cleverly Changing, the Parent 2 Parent initiative, and members of the sickle cell disease (SCD) community nationwide.

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.