Imara is discontinuing the clinical development of tovinontrine (IMR-687), its experimental oral therapy for sickle cell disease (SCD) and beta-thalassemia, based on interim data from two Phase 2b clinical trials. While the therapy was generally safe and well tolerated in adults with both inherited blood disorders, it resulted in…
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Endari, an oral therapy approved to treat sickle cell disease (SCD) has been added to the Florida Medicaid Preferred Drug List (PDL), its manufacturer Emmaus Life Sciences announced. The PDL, according to the Florida Agency for Health Care Administration (AHCA), is a listing of medications that are considered…
Until Teonna Woolford faced her own infertility after a haploidentical (half-match) bone marrow transplant, she hadn’t given’t much thought to the lack of awareness and resources for sexual health across the sickle cell disease (SCD) community. Hospital stays, liver failure, and a hip replacement surgery were already part…
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…
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