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.

Large language models may offer complementary value to human expert analysis in the diagnosis of achondroplasia and other skeletal dysplasias.

A new study conducted in India has revealed that 40% of antenatal patients experienced severe fetomaternal hemorrhage, which can increase the risk of HDFN.

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

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 ...

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 ...