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…
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…
The U.S. Food and Drug Administration has granted orphan drug status to Sanofi’s rilzabrutinib, an investigational therapy aimed at preventing painful vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). This designation intends to encourage companies to develop therapies for rare diseases, or those affecting fewer…
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…
Sickle cell disease (SCD) is associated with enhanced immune B-cell responses and the production of self-reactive antibodies that target and destroy red blood cells, according to data from a mouse model of the disease and patient samples. These changes in immune responses were regulated by type-1 interferons (IFN-1), an…
The first patient has been enrolled in a Phase 1 clinical trial evaluating the safety and tolerability of Rigel Pharmaceuticals’ fostamatinib — a medication approved in the U.S. for treating an autoimmune condition — in people with sickle cell disease (SCD). The Phase 1 study (NCT05904093) is…
Immvention Therapeutix has partnered with Novo Nordisk to develop oral treatments for people with sickle cell disease (SCD) and other chronic conditions. The U.S.-based Immvention called the deal “a strategic collaboration and license agreement” in a company press release. “We are excited to partner with Novo…
Cognitive behavioral therapy (CBT) and pain education, each delivered using digital mobile systems and supported by a peer health coach, were equally effective at lessening the impact of sickle cell disease (SCD) pain, a study found. The U.S.-based clinical trial, CaRISMA (NCT04419168), involved more than 300 adults with…
Adding cyclosporin H to a new lentivirus-delivered gene therapy with enhanced anti-sickling properties boosted the uptake of genetic material in blood stem cells from newborns at risk of sickle cell disease (SCD), a preclinical study reports. With more genetic material inside stem cells, the therapy generated therapeutic levels of…
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