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

Total joint arthroplasty may be a safe and durable treatment option for patients with achondroplasia, a form of skeletal dysplasia.

The pathophysiology of Duchenne muscular dystrophy (DMD) revolves around the lack of functional dystrophin, which results in a fragile, permeable cell membrane and the dysregulation of calcium ...

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 ...

Spesolimab demonstrated rapid and effective disease control in a patient with generalized pustular psoriasis (GPP) undergoing hemodialysis.

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.

Patients with antiaquaporin 4 antibody-positive NMOSD and MOGAD often also have other comorbidities, a study found.

US Food and Drug Administration grants Orphan Drug Designation to VAS-101 for patients with sickle cell disease (SCD).

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

Use of older prophylactic agents such as danazol and tranexamic acid has markedly declined in Hungary, while on-demand-only management is increasingly common. Modern prophylactic therapies for ...

Postnatal anti-D immunoprophylaxis is ineffective in fully preventing alloimmunization and should thus be administered early in pregnancy.