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A naturally-occurring protein called hemopexin, also an investigational therapy for acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), safely reduced the signs of such crises in cell and animal models, a study has demonstrated. These findings support an ongoing Phase 1 trial (NCT04285827), sponsored by the…

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

Note: This story was updated March 29, 2022, to remove a reference to Phi Beta Sigma as the oldest honor society in the nation. Phi Beta Kappa is the nation’s oldest honor society. The Sickle Cell Disease Association of America (SCDAA) and the Phi Beta Sigma Fraternity…

The United Arab Emirates (UAE) Ministry of Health has approved Endari (L-glutamine) for sickle cell disease (SCD), according to Emmaus Life Sciences, the therapy’s developer. During the five-month review process, which was originally expected to take up to one year, the oral therapy was available by early…

The University of Mississippi Medical Center (UMMC) will use a portion of a $169,500 grant from the UnitedHealthcare Community Plan of Mississippi on sickle cell disease (SCD) treatment and provider training. The funding, which will also support UMMC’s efforts to address pediatric behavioral health issues throughout Mississippi, is…

Dosing will soon start in a Phase 1/2 trial of GPH101, an experimental gene-editing therapy designed to correct the genetic mutation that causes sickle cell disease (SCD). The first participant had been enrolled in the study late last year, but due to the recent surge of the SARS-CoV-2…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

Blood donation restrictions in England that had prevented at least 12,000 Black donors from giving blood were lifted by the country’s government late last year in what the Sickle Cell Society called “a landmark change.” Now, citing “an urgent need” in the U.K. for blood donation from Black donors…

The GBT Foundation is calling for proposals for its Access to Care Empowerment for Sickle Cell (ACE) grant program, which will provide up to $250,000 to community-based sickle cell disease (SCD) organizations to accelerate development of care access programs for SCD patients. The ACE program, which was established this…

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…