Healthcare professionals generally agree that genetic testing can be useful in the HAE diagnostic process, but many urge caution in interpreting results.

As of May 2025, 62 active clinical trials are ongoing for Fabry disease, including 25 interventional and 37 observational studies, of which 8 are patient registries. Among the trials with known ...

Researchers have established clinical indications for pediatric blood transfusions with various ABO/Rh blood types that emphasize Rh compatibility.

Elevated basal serum tryptase levels in SM and HαT, as well as overlapping clinical characteristics, complicate the diagnostic process for both disorders.

Frailty reduced the likelihood of CLL/SLL first-line treatment initiation during the CIT era, but the gap narrowed with novel therapies.

Long-term prophylaxis with lanadelumab has been linked to a marked reduction in attack rates in HAE, with the decline persisting in a real-world setting.

Fostamatinib has been found to be safe and effective in patients with heavily treated persistent/chronic immune thrombocytopenia (ITP).

Compound 23, a dual-target FGFR2/3 inhibitor, delivered superior growth improvements over infigratinib in achondroplasia mouse models.

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

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

There was high consensus among neurologists on treating acute attacks with high-dose steroids and plasmapheresis in severe cases. Rituximab is still the most prescribed treatment for neuromyelitis ...

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