People with sickle cell anemia have higher levels of mitochondrial DNA (mtDNA) — DNA specific to mitochondria, the cell’s powerhouses — circulating in the blood than healthy individuals, likely due to the abnormal retention of mitochondria in red blood cells, a study has found. Notably, patients’ mtDNA was found to…
News
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
Note: This story was updated June 16, 2021, to clarify that the HGB-207 and HGB-212 clinical trials were testing betibeglogene autotemcel, a gene therapy that uses the same viral vector as LentiGlobin. A patient diagnosed initially with the blood cancer myelodysplastic syndrome (MDS) after receiving LentiGlobin, Bluebird Bio’s investigational…
Imara has opened applications for its second-annual “Real Impact” grants for community-based nonprofit organizations that support people with rare blood disorders, such as sickle cell disease (SCD) and beta-thalassemia. Through this year’s initiative, the biopharmaceutical company is awarding up to $150,000 to U.S. organizations that seek to address…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
To inform sickle cell disease (SCD) patients and their caregivers about blood transfusions, which are commonly used to treat the disorder, the Sickle Cell Disease Association of America (SCDAA) is launching a set of educational materials. The pictorial booklet for children, and companion brochure for parents and caregivers,…
Treatment with oral Oxbryta (voxelotor) leads to rapid and sustained rises in hemoglobin levels, reduces red blood cell destruction (hemolysis), and improves overall health in adolescents and adults with sickle cell disease (SCD), according to full, nearly 1.5-year data from the Phase 3 HOPE clinical trial. The findings support the long-term…
A higher 600 mg daily dose of FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), was well-tolerated and safe, and improved several measures of red blood cell health after 14 days of treatment, according to…
Global Blood Therapeutics (GBT) has been granted exclusive global rights to two small-molecule research programs for sickle cell disease (SCD) as part of a newly announced agreement between GBT and Sanofi. The two early stage programs,…
The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the therapy and the upcoming Phase 1/2 trial — planned to start this summer — are the result of a…
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