Therapies for Gaucher disease generally fall into 2 main categories: enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Neither ERT nor SRT effectively treats the neurological ...

Comprehensive single-cell and spatial transcriptomic analyses of cholangiocarcinoma (CCA) uncovered distinct molecular programs distinguishing intrahepatic from extrahepatic subtypes, providing ...

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

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

Researchers developed a new clinical score to estimate the probability of NMOSD in patients presenting with optic neuritis.

Two phase 3 clinical trials are recruiting patients with CIDP to evaluate the efficacy of riliprubart treatment.

Rilzabrutinib shows improved platelet counts, reduced fatigue, and acceptable safety for patients with immune thrombocytopenia (ITP).

The CSHA Clinical Frailty Scale is a feasible tool for predicting treatment-related toxicity and discontinuation in older adults with CLL.

Primary humoral immunodeficiency (PI) is characterized by impaired antibody production and an elevated vulnerability to bacterial and viral infections. This article discusses approved subcutaneous ...

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

Understanding early metabolic patterns could help predict disease activity in patients with Huntington disease (HD), a study found.

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