FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, was found to be effective at raising the levels of fetal hemoglobin in healthy adults enrolled in a Phase 1 trial, according to new interim results. Study data also indicated that FTX-6058 was…
News
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
Several clinical research sites seeking to find new treatments for people with sickle cell disease (SCD) are joining a collaborative network created by the American Society of Hematology Research Collaborative (ASH RC). The initiative, called ASH RC Sickle Cell Disease Clinical Trials Network, has already engaged about 24,000 patients…
Since Sept. 21, 1983, when then-President Ronald Reagan’s proclamation created National Sickle Cell Anemia Awareness Month, advocates, patients, family members, doctors, and others impacted by sickle cell disease (SCD) have been pushing for increased research and awareness, and better treatments. This year will be no different, as virtual,…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
The Sickle Cell Disease Foundation (SCDF) will use a $1 million grant to educate sickle cell disease (SCD) patients living in the western U.S. about the COVID-19 vaccine and assist them in accessing vaccination programs. The goal is to increase the number of SCD patients in western U.S.
The Sickle Cell Consortium, a U.S.-based nonprofit, is hosting a virtual event in late August that will focus on facilitating conversations among people with sickle cell disease (SCD) and their caregivers. “We’re bringing these patients together to talk about what they’re experiencing, strategies for navigating the realities brought…
Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…
Imara has completed patient enrollment for its Phase 2b Ardent clinical trial, which is testing the safety and efficacy of IMR-687 for the treatment of sickle cell disease (SCD). “We are excited to have enrolled [participants] from across the world, including in Africa, making this a truly global…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
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