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…
Biopharmaceutical company Brooklyn ImmunoTherapeutics has launched a new center focused on research and development (R&D) of gene editing and cell-based therapies to treat cancer and rare blood disorders, including sickle cell disease. “Our new R&D facility will provide us with the ability to advance our research and development…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
Endari (L-glutamine), an approved oral treatment for sickle cell disease (SCD), has now been added to the Preferred Drug List in Texas. The move will make Endari more accessible to people in the state who are insured through Medicaid, the government program that provides health insurance to low-income…
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