IMR-687, an investigational therapy for sickle cell disease, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). It’s currently being evaluated in a Phase 2a trial in the U.S. and U.K. The Fast Track process is meant to get new therapeutics that treat…
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RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
Novartis‘ Solutions to Empower Patients (STEP) program will fund five nonprofit organizations that address some of the most pressing concerns in the sickle cell disease (SCD) community. Nearly $250,000 will be divided up among patient advocacy organizations and research institutions. Started in 2017, the STEP program funds innovative…
The Phase 2 STRONG-SCD clinical trial evaluating olinciguat as an investigational therapy for sickle cell disease is seeking participants. Olinciguat, being developed by Cyclerion Therapeutics (a spin-off of Ironwood Pharmaceuticals), is an investigational oral therapy that stimulates an enzyme called soluble guanylate cyclase (sGC), known to play a key…
In what’s viewed as a significant step toward addressing unmet needs in California’s sickle cell disease (SCD) community, scientists and policy leaders met recently to discuss proposed legislation, gaps in patient care, and to hear from patients. Called “The Impacts of Sickle Cell Disease in California Legislative Briefing,” the…
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…
Backed by a new study suggesting that sickle cell disease (SCD) is common among refugees in Southern Europe, some hematologists are calling for increased screening measures. Specialists are working to determine the extent of blood disorders within the population. They’re also outlining ways to help identify and treat…
Adults with sickle cell disease score worse than their healthy, unaffected siblings on cognitive tests, despite treatment with hydroxyurea, a study reports. The study “Neurocognitive functioning in symptomatic adults with sickle cell disease: A description and comparison with unaffected siblings” was published in the journal Neuropsychological…
The California Institute for Regenerative Medicine (CIRM) and the National Heart, Lung and Blood Institute (NHLBI) have entered into a “landmark” collaboration to co-fund and help speed the development of cell and gene therapies to cure sickle cell disease (SCD), according to a press release.
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Findings on Abnormal Hemoglobin Assembly Could Lead to Lower-Dose SCD Medications, Researchers Say
A study of sickle cell disease (SCD) at the molecular level found abnormal hemoglobin molecules assemble much faster and less efficiently than previously thought — findings that could lead to the development of new treatments. The researchers said better understanding of the assembly process is the first step toward…
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