Important gains were observed in key areas, including basic theory, transfusion compatibility testing, and clinical transfusion application. A structured 6-month fellowship program in transfusion ...
Approximately 85% of cases of MG are caused by autoantibodies targeting the acetylcholine receptor (AChR+), while 15% are antibody negative (AChR-). Patients with acetylcholine receptor ...
Anti-transferrin receptor (TfR) antibody-coupled acid-alpha-glucosidase (GAA) led to robust glycogen clearance and reduced neuroinflammation in the central nervous system of a mouse model of Pompe ...
Transthyretin amyloids-specific drugs led to a trend toward less decline in the 6-minute walk distance and were associated with a significantly blunted decline in Kansas-City ...
A comprehensive hematologic assessment can be useful in cases of CAD without common etiologies. “While CAD is most commonly linked to clonal B-cell disorders such as chronic lymphocytic leukemia and ...
Whole-genome resequencing used to assess ATP7B genotypes found a higher frequency of impaired finger tapping in patients carrying the p.P992L mutation. A variety of sociodemographic factors were found ...
Carolina Barnett-Tapia, MD, PhD, is an associate professor of neurology at the University of Toronto. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Carolina Barnett-Tapia, MD ...
Intravenous immunoglobulin-induced thrombocytopenia was reported in 146 cases, with the highest incidence seen in patients with immune-mediated skin diseases. Intravenous immunoglobulin (IVIg) therapy ...
Most participants remained bleed-free across both studies, and all reported bleeds were managed effectively. FRSW117, a novel investigational therapy for severe hemophilia A, showed promise as a ...
A baseline bleeding risk of 7.3 bleeds per year was observed without any FIX, and targeting trough FIX levels between 10 and 20 IU/dL reduced bleeds to very low or eliminated them. In people with ...
Kerrie Walters, who has MS, and her service dog, Wooldridge, at home in Grants Pass, Oregon (Credit: Jennifer Butler Photography) Kerrie Walters first heard of PoNS in a chance encounter with TV ...
Ruxolitinib exerts its activity independently of oncogenic Janus kinase 2 (JAK2)-V617F signaling in myeloproliferative neoplasms (MPNs) like myelofibrosis (MF), according to a new study published in ...
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