A CRISPR/Cas9-modified Janus kinase 2 (JAK2) V617F model may be useful to develop new therapies against myeloproliferative neoplasms (MPNs), showed a new study published in the International Journal ...

Among patients with severe hemophilia A and B, treatment with the monoclonal antibody marstacimab has been associated with reduced rates of bleeding, according to a systemic review and meta-analysis ...

Sheila Jacobs is the president of STJ Editorial Services. For more than 25 years, Sheila has been providing writing, fact-checking, and editorial services to numerous clients in the medical education, ...

There is significant variability in terms of management practices across Canadian medical centers in terms of red blood cell (RBC) and platelet transfusions in preterm newborns, according to a study ...

Risk factors for thymidine kinase 2 deficiency (TK2d) include inheritance patterns, molecular pathogenesis, genotype-phenotype correlations, and the risk of secondary complications after disease onset ...

Spesolimab is a therapy that works by targeting interleukin-36 receptor signaling. Its efficacy against generalized pustular psoriasis (GPP) was established in the Effisayil studies; these studies ...

Patients with Fabry disease appear to have a higher incidence of renal cell carcinoma than the general population, according to a recently published study in Molecular Genetics and Metabolism. “The ...

The US Food and Drug Administration (FDA) has granted a Priority Review designation to a New Drug Application (NDA) for TransCon ® CNP (navepegritide) for the treatment of children with achondroplasia ...

Sheila Jacobs is the president of STJ Editorial Services. For more than 25 years, Sheila has been providing writing, fact-checking, and editorial services to numerous clients in the medical education, ...

Caregivers and healthcare providers of patients with hemophilia both expressed unmet needs regarding information provision, a problem that may be remedied by using digital information platforms, ...

Patients with myasthenia gravis (MG) have a changed CD4 T cell phenotype, according to a new study published in the Journal of Neurochemistry. The authors of the study suggested that the frequency of ...

Patients with severe hemophilia A and B who develop inhibitors exhibit fundamentally distinct immune responses to replacement factor VIII and factor IX, as revealed by single-cell sequencing of ...