Results of the ApproaCH trial showed that navepegritide improved AGV over placebo in pediatric patients with achondroplasia.

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.

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

Patients with hepatocellular carcinoma (HCC) face a significantly elevated risk of thrombosis, with incidence higher in Europe.

In June 2024, the US Food and Drug Administration (FDA) approved Vyvgart ® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc; Argenx BV) for the treatment of adults with chronic inflammatory ...

The pozelimab and cemdisiran combination treatment demonstrated positive phase 3 results for patients with PNH.

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Claire Booth, MBBS, PhD, cofounder of the AGORA Initiative.

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

An estimated 100,000 Americans have sickle cell disease, the vast majority of them of African or Caribbean origin. But more than 1.5 million Black men in the US are genetically predisposed to a much ...

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

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with DMD, a study found.