Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Sithara Ramdas, MD, of Oxford Children’s Hospital in England, on neonatal and juvenile myasthenia gravis.

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

Physical activity before or during pregnancy was not associated with negative maternal/neonatal outcomes; however, physical activity was not statistically significantly associated with positive ...

In patients with the granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) subtypes of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) who have kidney ...

A patient with KIT-C -negative systemic mastocytosis (SM) initially presented with severe anemia alongside intraosseous-intramuscular hemorrhage in a case report recently published in Cureus. Atypical ...

Vosoritide demonstrated sustained long-term efficacy and safety in children with achondroplasia, significantly improving growth outcomes and maintaining high treatment adherence in a real-world ...

Tailored, risk-stratified rituximab regimens including yearly infusions may offer sustained disease control in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), ...

Elastase-inhibitory activity (EIA) is associated with chronic obstructive pulmonary disease (COPD) in patients with alpha-1 antitrypsin deficiency (AATD), according to a study published in PLOS One.

In patients with eosinophilic granulomatosis with polyangiitis (EGPA), a rare form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), adding cyclophosphamide to glucocorticoid ...

There were no significant changes in the levels of a polysialic acid (polySia), a glycoepitope that plays a key role during neuronal development and plasticity, in chronic inflammatory demyelinating ...

Which of the statements below about the differential diagnosis of immunoglobulin G4-related disease (IgG4-RD) are correct? Select one of the options for each question, then submit your answers to see ...

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 ...