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…
Novartis and the Bill and Melinda Gates Foundation are collaborating to develop a single-shot gene therapy that would cure sickle cell disease (SCD) without the need to engineer cells outside the body. The partnership aligns with Novartis’ efforts to better treat SCD, and the Gates Foundation‘s…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Bluebird Bio has paused two clinical trials of LentiGlobin, its gene therapy candidate for sickle cell disease (SCD), after learning that a patient who received treatment more than five years ago now has developed acute myeloid leukemia (AML), a type of blood cancer. In line with study protocols,…
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