Age over 75 years was significantly associated with reduced survival and shorter time on treatment. Ibrutinib remains an effective therapy for chronic lymphocytic leukemia (CLL), but older patients ...

Expert consensus has been achieved on a minimum data set for growth hormone monitoring in children with Prader-Willi syndrome (PWS).

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.

General anesthesia provides similar pain control to nerve block-assisted anesthesia after limb lengthening surgery, common in achondroplasia.

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

Models of clinical score trajectories were developed using natural history data from patients with HD and tested against placebo response.

Senior correspondent Larry Luxner interviews Chris Lewis, whose father, comedian Jerry Lewis, was a tireless advocate for people with muscular dystrophy.

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

Study highlights rhe urgent need for a national consensus and standardized care guidelines in Brazil to improve outcomes for patients with gneralized pustular psoriasis (GPP).

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