Given the results, the investigation of intranasal carbetocin in PWS has been halted. Intranasal carbetocin did not demonstrate clinical benefit for hyperphagia in patients with Prader-Willi syndrome ...
The levels of NT-proBNP, left ventricular mass index, maximal wall thickness, and E/E′ ratio were higher in patients with worse neurological stages compared to those in the early neurological stages ...
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 ...
The mean gestational age at delivery was significantly higher in the late intrauterine transfusion group than in the early intrauterine transfusion group (P .001). Late intrauterine blood transfusions ...
The evaluation of the cerebrospinal fluid and imaging techniques, like high-resolution ultrasound, can improve diagnostic certainty. A new article that appeared in the scientific journal Neurologic ...
In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...
Immunohistochemistry can identify concomitant SM in acute myeloid leukemia, leading to better risk stratification and management. The most common forms of AML in pediatric patients are those ...
Patients who had a poor outcome had a higher GLOBE score and UK-PBC risk score compared to patients with good outcomes after 1 year of treatment with fenofibrate add-on therapy. The GLOBE and UK-PBC ...
The RAPIDe-3 trial is evaluating an oral bradykinin B2 receptor antagonist in approximately 120 participants with hereditary angioedema. Pharvaris has updated its guidance, indicating that topline ...
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 ...
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 ...
The primary outcome measures include assessing I-RODS scores at baseline and monitoring variations at 6 and 12 months after enrollment An upcoming observational, noninterventional study aims to assess ...