News

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…

Naproxcinod, an investigational therapy being developed by Fera Pharmaceuticals as a treatment for sickle cell disease (SCD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). Orphan drug status provides regulatory support and financial benefits to experimental treatments for diseases that affect fewer…

An enzyme known to play a key role in blood vessel health is present at low levels in the cells lining the lung blood vessels of patients with sickle cell disease (SCD), which may affect how these cells stick to their supporting matrix, a study has found. Lack of…

Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…