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.
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…
A newly developed treatment approach, known by the acronym SICKLE, was shown to be a safe strategy to manage and treat long-lasting sickle cell disease-related ulcers in a case report. This holistic approach was able to heal skin wounds, which had not responded to prior standard treatments, within six months. The…
More people search for information on sickle cell disease (SCD) during winter than summer, indicating seasonal changes in symptom flare-ups, an analysis of Google search queries shows. Findings were published in the study, “Seasonal manifestations of sickle cell disease activity,” in the journal Nature Medicine. In SCD,…
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…
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