Assessing Epidemiological Data Points The findings from this study echo that of another by Trivella and colleagues, also published in Hepatology Communications. This 2023 study is a literature review ...

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 ...

Adult patients with Fabry disease have a lower lean mass compared to healthy people, according to a new study published in the Journal of Rare Diseases. The accumulation of lyso-Gb3 is also correlated ...

Updated guidelines for managing patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) from the British Society for Rheumatology (BSR) were published recently in ...

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 ...

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 ...

A new case has been reported involving the successful sedation of a patient with cold agglutinin disease (CAD) for an urgent gastroscopy procedure. The report, published in Cureus, reviewed several ...

Patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) who were treated with tafamidis for a year showed stabilization of several cardiac biomarkers, according to a study published in ...

Researchers presented the case of a patient with chronic inflammatory demyelinating polyneuropathy (CIDP) who experienced syncope and new-onset bifascicular block while being treated with intravenous ...

Enrollment is under way to recruit patients for a phase 3 study designed to assess intravascular (IV) treatment with zaltenibart among individuals with paroxysmal nocturnal hemoglobinuria (PNH), ...

DALLAS—An atmosphere of grave concern—if not outright panic—is gripping the rare disease community as massive budget cuts and layoffs at the US National Institutes of Health (NIH) endanger tens of ...

DALLAS—A 16-year-old boy with Duchenne muscular dystrophy (DMD) died of acute liver failure several months after receiving delandistrogene moxeparvovec-rokl (Elevidys ®), a gene therapy developed by ...