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

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

A new dual-feature framework was able to increase the diagnostic ability of myeloproliferative neoplasms, including MF, a study found.

The results of the current meta-analysis reinforce the safety and efficacy of marstacimab in reducing annualized bleeding rates among patients with severe hemophilia A and B.

A recent study found that the Pettersson scored retained its value in assessing radiographic damage, especially among pediatric patients with hemophilia.

Patients with myasthenia gravis (MG) have a changed CD4 T cell phenotype, according to a new study published in the Journal of Neurochemistry. The authors of the study suggested that the frequency of ...

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.

Neurodevelopmental and mental disorders may be common in patients with DMD, and some may require pharmacological treatment, a study found.

Patients with Fabry disease appear to have a higher incidence of renal cell carcinoma than the general population, a study found.

The FDA approved garadacimab-gxii, the first once-monthly prophylactic treatment of HAE to inhibit the disease cascade at its source.

The FDA granted Priority Review status for TransCon CNP for achondroplasia based on positive early results of significantly improved AGV.

There may be a higher prevalence of hereditary angioedema, according to a US insurance claims-based data analysis.