In cellular models, curcumin affected several HD-related pathways, suggesting it may be useful as a tool for studying disease ...

Positive levels of circulating anti-nephrin autoantibodies increase the risk of FSGS recurrence after kidney transplant.

A report of 2 patients Dravet syndrome (DS) who developed ARDS suggests it may be an underrecognized extraneurologic feature ...

A sports coach and athlete explains how living with Pompe disease has changed his perspective on the purpose of exercising.

In the first report of its kind, a patient with HD and depression who received esketamine experienced rapid but short-lived improvement.

Researchers have validated 45 mg/kg of intravenous nipocalimab per week as the optimal regimen for the phase 3 AZALEA trial.

In a real-world study, nearly half of adults with LGS, DS, or TSC who added CBD experienced a 50% or greater reduction in ...

Bezuclastinib produced significantly greater symptom improvement than placebo at 24 weeks in adults with symptomatic ...

Growing evidence suggests viral infections can exacerbate myasthenia gravis (MG) or even contribute to its development.

Severe fatigue remains common among patients with PNH receiving long-term eculizumab and appears to stem from more than anemia alone.

Two cases of SM with associated hematologic neoplasm demonstrated how clinical severity, molecular findings, and treatment ...

Turkish patients with genetically confirmed PMDs are still likely to be managed with empirical vitamin and cofactor ...