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 ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

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 ...

Bone marrow flow cytometry is one of the most efficient ways to diagnose or exclude PNH. A patient with a history of cortical vein thrombosis with new-onset pancytopenia was diagnosed with paroxysmal ...

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 ...

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 ...

PHOENIX, Arizona—Biomarkers “are a hot topic in myasthenia gravis (MG),” Anna Punga, MD, PhD, said during her keynote speech at the Myasthenia Gravis Foundation of America (MGFA) session of the 2023 ...

The novel anti-IL-36 receptor monoclonal antibody recibokibart demonstrated favorable safety and efficacy in treating patients with acute generalized pustular psoriasis (GPP) in a phase 1b study ...

Imagine getting the chance to eat your favorite meal, but on your first bite, you struggle to chew, and your eyes suddenly water as the food gets stuck in your throat as you swallow. Struggles with ...

Approximately 42% of patients showed evidence of SMN1 to SMN2 conversion, confirmed by the presence of SMN1-specific variants downstream of SMN2. To shed more light on the genetic determinants of SMA ...

The severity of clinical manifestations in CAD can vary depending on the specific type of cold-sensitive antibodies involved. While cold agglutinin and Donath-Landsteiner antibodies recognize antigens ...

Patients with CIDP had significantly lower levels of total N-glycans in their serum compared to healthy people. The levels of sialylated N-glycans in the serum could be a novel biomarker in chronic ...