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

The expression of AQP4 In the respiratory epithelium suggests that lung involvement might be an unrecognized feature of NMOSD. Patients with neuromyelitis optica spectrum disorder (NMOSD) should be ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of CIDP is now open.

Some disease-specific PROMS are moderately recommended in the monitoring of QoL in patients with HAE, but more validation is needed.

In observance of Rare Disease Day, Rare Disease Advisor has curated this collection of content to illustrate the issues facing the rare disease community, highlight the advocacy work being done in the ...

Bilirubin results may differ between neonates of different ethnicities, a possibility that should inform the interpretation of these results.

Primary interosseous Rosai-Dorfman disease does not belong to the IgG4-RD spectrum, according to a recent study.

A CRISPR/Cas9-modified Janus kinse 2 V617F model may be useful to develop new therapies against myeloproliferative neoplasms.

The appendicular lean mass was lower in patients with Fabry disease at a mean of 20,324 g compared to 25,529 g in healthy people.

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings.

Patients with chronic lymphocytic leukemia (CLL) receiving FCR chemotherapy may be at greater risk of other malignancies.

Cyclophosphamide prophylaxis for graft versus host disease after allogeneic hematopoietic stem cell transplantation in MF is very effective.