Top-line data from the Phase 3 part of RISE UP, a clinical trial testing mitapivat in people with sickle cell disease (SCD), are expected by the end of the year, according to developer Agios Pharmaceuticals. If those results are positive and mitapivat wins regulatory approval for SCD, it…
News
Hydroxyurea, an approved medication to reduce the frequency of pain crises associated with sickle cell disease (SCD), may ease sleep-disordered breathing in children, perhaps by reducing inflammation, according to a study conducted at St. Jude Children’s Research Hospital, in Memphis. “Hydroxyurea therapy was associated with improved breathing…
An ongoing, early-phase clinical trial of pociredir, an oral small molecule being developed by Fulcrum Therapeutics as a possible treatment for sickle cell disease (SCD), is still recruiting adult patients at locations in the U.S. and one in South Africa. This Phase 1b trial, called PIONEER (NCT05169580),…
The use of computed tomography pulmonary angiography (CTPA) to assess the presence of blood clots in lung blood vessels increased significantly in people with sickle cell disease (SCD) after 2020, a study in Bahrain shows. The study indicates that a third of patients underwent several scans, many of which…
More perceived social support from classmates is associated with less disease severity among children and adolescents with sickle cell anemia, the most common and often the most severe form of sickle cell disease (SCD), according to a new study from researchers in Nigeria. Social support from parents also was…
Long-term treatment with hydroxyurea reduces emergency department visits and days spent in the hospital for children with sickle cell anemia, the most common and usually the most severe form of sickle cell disease (SCD), a real-world study has found. Consistent benefits among pediatric patients given the approved…
Vascarta is launching a proof-of-concept Phase 1 clinical study to test its topical curcumin gel VAS-101 — which uses a component of turmeric — in people with sickle cell disease (SCD). The experimental therapy, also known as Vascepto, is designed to be applied onto the skin. It contains curcumin,…
The U.S. Patent Office has given AB Science notice that it will allow a patent to be issued covering the use of the experimental therapy masitinib for sickle cell disease (SCD). This medical use patent will be valid until November 2040, giving AB Science exclusive rights to develop masitinib…
Women with sickle cell disease (SCD) have markedly elevated levels of C-reactive protein (CRP), a marker of body-wide inflammation, during the first half of their menstrual cycles, and these variations may contribute to recurrent patterns of vaso-occlusive events (VOEs), complications resulting from blood vessel obstruction that include painful…
Researchers have developed a modelling system of tiny blood vessels to track inflammation-driven blood clot formation that can cause complications in sickle cell disease (SCD) and other conditions. “One of the biggest things about being able to create [blood clots] under inflammatory conditions is that we are now able…
Recent Posts
- Attending graduate school with sickle cell disease requires balance
- New off-the-shelf gene editing may treat sickle cell without transplant
- SCD cure rate hits 95% with new stem cell transplant procedure
- After a stressful time away, a return to my version of normal is on the horizon
- Developer seeking FDA’s accelerated approval for mitapivat for SCD
- Intense crises: When sickle cell pain becomes unbearable
- Newer blood thinners tied to less bleeding in sickle cell disease study
- New reimbursement deal in Germany expands access to Casgevy for SCD
- New York study finds regional gaps in sickle cell disease hospitalizations
- When a sickle cell pain crisis interrupts a stretch of good health, part 1