AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
News
Emmaus Life Sciences has submitted an application seeking the approval of its oral medication Endari (L-glutamine) to treat sickle cell disease (SCD) in Kuwait. The company announced that the Kuwait Drug and Food Control administration, which is responsible for registering pharmaceutical products in the country, has accepted…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
As a stem cell transplant is the only curative treatment for sickle cell disease (SCD), a blood disorder that disproportionately affects Blacks and African Americans, “Cheek Week” is again calling for the participation and inclusion of donors from different ethnic backgrounds in the Be The Match registry,…
A new research collaboration between Jasper Therapeutics and Aruvant Sciences will evaluate the use of JSP191, Jasper’s anti-CD117 monoclonal antibody, as a conditioning agent for ARU-1801, Aruvant’s experimental gene therapy for sickle cell disease (SCD). “The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy…
A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.
Note: This story was updated July 7, 2021, to clarify that Hertz Nazaire lived with his godmother, rather than his grandmother, after his mother’s death. A Connecticut-based artist living with sickle cell disease and blindness in his right eye is using his art to advocate for…
Velvet Brown-Watts was devastated when her son, Jeremiah Watts Jr., now 16, was diagnosed with sickle cell disease in 2004 at 2 months old. “It was probably one of the worst things I ever heard,” Brown-Watts, 46, told Sickle Cell Disease News. Doctors at…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
To improve life for people with sickle cell disease (SCD), Global Blood Therapeutics (GBT) has awarded grants worth $50,000 each to nine community-based, nonprofit groups and institutions across the U.S. to speed the development and use of sustainable, access-to-care programs. These one-year grants are part of GBT’s Access to…
Recent Posts
- Tips for enjoying the most wonderful time of the year with sickle cell
- Big Nova donates $3M to expand access to stem cell transplants
- Investigational SCD therapy shows stronger results at higher dose
- Risto-cel showing lasting benefits for people with severe SCD in trial
- The price of living with a physical disability in a non-inclusive society
- Casgevy safely prevents sickle cell crises in children: Trial data
- Curcumin skin gel shown to improve heart health in SCD mice
- Socioeconomic status not linked to stroke risk in children with SCD
- Weathering the stages of denial before reaching acceptance
- New Aflac children’s book supports youngsters with SCD, cancer