The Centers for Medicare & Medicaid Services (CMS) has announced that 33 states, along with the District of Columbia and Puerto Rico, have agreed to participate in the Cell and Gene Therapy (CGT) Access Model, which is intended to boost access to cell and gene therapies for people with…
News
Acute chest syndrome (ACS), a form of lung injury associated with sickle cell disease (SCD), is driven by the abnormal activation of a part of the immune system known as the complement cascade, a study found. The researchers found elevated complement activation in SCD patients, especially in those…
Fentanyl may be a feasible, safe, and effective option when administered intranasally (into the nose), to manage acute moderate to severe pain episodes in adults with sickle cell disease (SCD), a study in Canada reports. For faster and non-invasive pain relief, it has been widely used in children with…
A camp in North Carolina offers children living with sickle cell disease the chance to build confidence, independence, and emotional strength while connecting with others who also live with a chronic blood disease. Each summer, Camp Hope provides kids with sickle cell to learn about themselves while participating…
The investigational gene-editing therapy OTQ923 resulted in normalization of the speed of blood flow in the brain of three adults with sickle cell disease (SCD), according to data from a Phase 1 clinical trial. Findings suggest the treatment could help lower the risk of stroke in patients. The…
The first healthy volunteer has been dosed in a Phase 1 clinical study of CLY-124 — a novel oral small molecule that Cellarity is developing to treat sickle cell disease — following clearance of the company’s investigational new drug application by the U.S. Food and Drug Administration. “Initiation…
Higher pain scores at the time of discharge from the emergency department (ED) may predict the likelihood of hospitalization in children with sickle cell disease (SCD) who seek emergency care for acute pain episodes, according to a study in the U.S. The study also found that patients with severe…
Adults with sickle cell disease (SCD) who are started on buprenorphine to ease chronic pain — either in a clinical setting or at home via telemedicine — may be able to reduce their use of opioids and visit the hospital less frequently, a small study suggests. Buprenorphine induction —…
Most people with sickle cell disease (SCD) seem to meet or exceed current physical activity recommendations for the general population, a recent study in Senegal shows. Notably, greater step counts and time spent in more intense activities were associated with lower pain frequency and intensity, as well as lower…
BEAM-101, a one-time, gene-edited cell therapy, continues to show durable therapeutic benefits more than one year after treatment in people with sickle cell disease (SCD), according to new data from the Phase 1/2 BEACON trial. The treatment’s developer, Beam Therapeutics, shared data from the first 17 patients dosed…
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