Global Blood Therapeutics (GBT) announced the five organizations that will receive more than $200,000 worth of grants through its new Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL). ACCEL was launched in February by the clinical-stage biopharmaceutical company with the goal of improving the…
News
Prominent sickle cell advocate and philathropist Howard Woolley has won a Sickle Cell Outstanding Service Award, presented recently at the 2019 Roland B. Scott Memorial Symposium. Themed “Preparing the next generation of advocates for sickle cell disease healthcare,” the symposium, held at Howard University, featured a…
Robert and Tanique Mitchell still remember the moment they found out that their granddaughter, Kiarra Roseburgh, would be born with sickle cell disease (SCD). “We realized that we needed to prepare, and we made two goals as a family: to provide emotional support for Kiarra and to empower ourselves…
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…
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…
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