News

A Phase 2b clinical trial that is investigating the safety and effectiveness of IMR-687 in people with sickle cell disease (SCD) has dosed its first participant, according to the potential therapy’s developer, Imara. The study, Ardent (NCT04474314), intends to enroll almost 100 adults who have SCD. Recruitment…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

The Scottish Medicines Consortium (SMC) has approved the use of Xromi (hydroxycarbamide), a liquid form of hydroxyurea, for treating children with sickle cell disease (SCD). Xromi is expected to be used in children older than 2 and younger than 9 who…

The cannabis company Parallel, which aims to improve quality of life with its products, has launched a 10-year collaboration with the University of Pittsburgh, known as Pitt, to investigate the potential of medical marijuana for treating people with sickle cell disease (SCD). Pitt’s research program will receive an…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

The National Heart, Lung, and Blood Institute (NHLBI) awarded nearly $1 million to Athena Starlard-Davenport, a professor at the University of Tennessee Health Science Center (UTHSC), to support work into fetal hemoglobin as a potential way of treating sickle cell disease (SCD). “For the past 100 years,…

With a stem cell transplant (SCT) being a potentially curative treatment for sickle cell disease (SCD), a blood disorder that disproportionately affects Blacks, a donor registry is encouraging more African-Americans to join its ranks. Be The Match, the world’s largest and most diverse marrow registry, has opened…

Emotional distress affected nearly half of the parents caring for adolescents with sickle cell disease in a recent study, with greater numbers of hospital visits and other life disruptions predicting higher stress levels. Psychosocial support and education to help caregivers be more resilient and better able to cope could improve…

A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for the prevention of vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the conditional approval of…

Most people with sickle cell disease (SCD) in the U.S. are satisfied with their primary care practitioners, but less so with emergency care given at a hospital to manage severe pain, according to a survey of more than 400 teenage and adult patients. How a person expects to be…