News

A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell disease (SCD) by successfully editing genes in stem cells within the bone marrow of mice. Data from this proof-of-concept study…

A spoken word poetry contest is welcoming submissions from people with sickle cell disease (SCD) and their caregivers. The contest, “Lift Every Voice to Shine the Light on Sickle Cell,” is being jointly launched by Global Blood Therapeutics (GBT) and the Sickle Cell Disease Association of America…

The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to include patients ages 3 and older who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The therapy was first approved…

The U.S. Food and Drug Administration has cleared the way for a Phase 1 trial of HBI-002, Hillhurst Biopharmaceuticals‘ oral carbon monoxide treatment for sickle cell disease (SCD). The trial will assess the safety, tolerability, and pharmacokinetics (how the therapy moves through the body) of single and multiple…

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…

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,…