Rapid and repeated imaging of blood flow through the eyes can inform physicians of a patient’s risk of sustaining eye damage and developing vision problems related to sickle cell disease (SCD), as well as of how effective a given therapy is, a recent study shows. According to researchers, this…
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…
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…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
The European Commission and the U.S. Food and Drug Administration have granted orphan drug status to CSL889, an investigational therapy for the treatment of acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), CSL Behring, the medicine’s maker, announced in a press…
Siklos (hydroxyurea) — indicated for the treatment of children 2 and older with sickle cell disease — is now available in 100 mg scored tablets, in addition to the 1,000 mg triple-scored tablet form. Scored tablets make measuring medicine more flexible, giving patients greater control in finding…
The Bill & Melinda Gates Foundation has awarded a $3.3 million grant to GreenLight Biosciences to develop gene therapies revolving around messenger RNA (mRNA) — the intermediate molecule between DNA and proteins — for sickle cell disease (SCD)…
Genetic analyses of African patients identified several genes and biological pathways that are associated with disease severity and longevity, and that might serve as new therapeutic targets in sickle cell disease (SCD). The study, “Genetic modifiers of long‐term survival in sickle cell anemia,” was published in…
Microbes in the gut play a key role in inducing vaso-occlusive crises (VOCs) in response to stress, a study in mice suggests. Stress is known to trigger VOCs in people with sickle cell disease (SCD) and inflammation in those with cardiovascular disease, but in ways not fully understood.
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
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