Gene expression in blood from an individual with β‐thalassemia: An RNA sequence analysis
ConclusionWe compare our findings with published results of RNA ‐sequencing analysis of sickle cell disease and erythroblasts from a KLF1‐null neonate with hydrops fetalis, and recognize similarities and differences in their transcriptional expression patterns.
The company's crizanlizumab will receive a priority review, shortening the standard FDA process from ten months to six months.
Novartis today announced the US Food and Drug Administration (FDA) accepted the company's Biologics License Application (BLA) and has granted Priority Review for its investigational sickle cell medicine crizanlizumab (SEG101). If FDA-approved, crizanlizumab is expected to represent the first monoclonal antibody targeting the P-selectin mediated multi-cellular adhesion in sickle cell disease.
Researchers at Tufts University and the Chinese Academy of Sciences have developed a new lipid nanoparticle which can deliver CRISPR/Cas9 gene editing tools into organs with high efficiency, suggesting that the system is promising for clinical applic...
CONCLUSION: Transition to adult providers is predicted by establishing care with an adult SCD provider within 6 months of transition and being on pre-transition disease-modifying therapy. Transition may be improved if pediatric hematology centers assist and verify adult provider contact within 6 months of transition and engage patients of all disease severity during transition. PMID: 31307033 [PubMed - as supplied by publisher]
Authors: Patel DA, Akinsete AM, Connelly JA, Kassim AA Abstract Introduction: Severe sickle cell disease is associated with progressive end-organ damage and early mortality in adults. While allogeneic hematopoietic cell transplant from a matched related donor is curative, the vast majority of patients do not have a compatible sibling. Accordingly, platforms using haploidentical donors have been developed, which provide near universal availability. Areas covered: This review focuses on the two commonly used approaches for haploidentical hematopoietic transplants, namely T-cell deplete and T-cell replete, each of whi...
Conclusions:The prevalence of elevated PLMI in children with SCD was higher than in previously published data. Elevated PLMI was significantly associated with greater rates of cerebrovascular disease as detected by MRI.Citation:Lin J, Morrone K, Manwani D, Chernin R, Silver EJ, Shifteh K, Sin S, Arens R, Graw-Panzer K. Association between periodic limb movements in sleep and cerebrovascular changes in children with sickle cell disease.J Clin Sleep Med. 2019;15(7):1011–1019.
No abstract available
Conclusion: This exploratory study found an enhanced inflammatory response to cholecystectomy in patients with SCA compared with patients with HbAA. Minimally invasive surgical strategies for this patient group may help to mediate this response. PMID: 31285651 [PubMed - in process]
Conditions: Sickle Cell Disease; Neuropathic Pain Intervention: Sponsors: The University of The West Indies; Avicanna Inc Recruiting
[Ghanaian Times] Director General of the Ghana Health Service, Dr.Anthony Nsiah-Asare has said, centres of excellence for the treatment of sickle cell will be established across the country to fight the disease.