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 prevalence of PBC is expected to rise in the United States and the wider world, requiring concrete, proactive action.

There are several experimental therapies under investigation for the treatment of Fabry disease, including enzyme replacement therapy (ERT), substrate ...

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.

Which of the statements below about the histology of hemolytic disease of the fetus and newborn (HDFN) are correct? Select one of the options for each question, then submit your answers to see how ...

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 ...

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

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

Keyana Sullivan, diagnosed with Pompe disease, uses a wheelchair and is dependent on a ventilator. Nonetheless, she is very goal-oriented. Keyana is a creative writer with a bachelor's degree in ...

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