News

Adding Cell Source’s special class of immune cell, Veto T-cells, to blood stem cells from an unrelated donor led to a successful transplant and to the production of normal red blood cells in a mouse model of sickle cell disease (SCD), the company announced. The preclinical Cell Source-sponsored…

Global Blood Therapeutics (GBT) has opened its grant program — the Access to Excellent Care for Sickle Cell Patients (ACCEL), now in its third year — to proposals supporting programs aiming to improve access to high-quality healthcare for people with sickle cell disease (SCD). GBT will donate up…

Registration is open for the 8th annual “Walk with the Stars,” one of the largest fundraisers organized by the Sickle Cell Disease Association of America (SCDAA) that supports the development of better treatments and a cure for sickle cell disease (SCD). All proceeds from the July 10 event…

LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), is unlikely to be the cause of a blood cancer that arose in one of the patients who received the therapy in an early clinical trial, according to new analyses. In February, Bluebird paused two clinical…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

The opioid crisis, along with the stigma of suspected drug use, and racial bias all contribute to make treatment access more difficult for people with sickle cell disease (SCD). That was the take-home message of the…

Vascular electrical stimulation therapy (VEST), a form of local electrical stimulation applied to the skin, can lower the duration of painful vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD), according to data from a Phase 2 clinical trial. Based on these findings, study investigators argue that…

The quality of life of people with sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT) — two inherited blood disorders — tends to improve if they receive a stem cell transplant or gene therapy, a review study  found. The study, “A systematic review of quality…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…