Biomarkers are increasingly recognized for their use in diagnosing and tracking disability progression in NMOSD.

Diet and nutrition play a key role in managing Duchenne muscular dystrophy (DMD). Dietary goals include preserving lean muscle mass, preventing weight gain and fat accumulation, maintaining bone ...

Monomeric C-reactive protein (mCRP) levels were significantly elevated in AAV and declined earlier than pentameric CRP during treatment.

An extension of the dosing interval of lanadelumab retains effectiveness in reducing attack frequency in patients with HAE.

In those with HAE-C1NH, whole blood under cold activation demonstrated a noticeable elevation in bradykinin levels, with minimal impact on healthy subjects.

Investigational gene therapy showed sustained clinical benefit and long-term safety in severe hemophilia B over 13 years of follow-up.

Adding cyclophosphamide to glucocorticoids did not reduce relapse risk at 12 months in EGPA patients without poor prognosis factors.

ATTR treatments and aortic valve replacement are associated with significant survival benefits in patients with aortic stenosis and ATTR-CM.

Vosoritide can improve growth outcomes in children with achondroplasia when administered continuously from infancy to final adult height.

Diazoxide choline controlled-release may significantly enhance quality of life for individuals with PWS and their families.

Models of clinical score trajectories were developed using natural history data from patients with HD and tested against placebo response.

Mutations in the DNMT3A gene are an independent risk factor for arterial thrombotic events in patients with myelofibrosis (MF), a study found.