Vosoritide therapy led to remarkable improvements in the mechanical axis angle and mechanical lateral proximal femoral angle in the younger patients. One year of treatment with vosoritide has been ...

Patients, families and staff gather for a group photo during the 2023 Dystroglycanopathies Patient & Family Conference (Photo courtesy of the University of Iowa Wellstone Center) Katherine Mathews, MD ...

Which of the statements below about the prognosis of generalized pustular psoriasis (GPP) are correct? Select one of the options for each question, then submit your answers to see how well you scored ...

The study identified 412 patients with the ATTR-mixed phenotype. Integrating natural language processing, machine learning, and explainable artificial intelligence (AI) may help in the timely ...

(Getty Images) Because of the significant disease burden of CLL, many patients struggle with issues related to health-related quality of life. In recent years, the clinical emphasis on quality of life ...

Approximately 31% of dermatologists reported difficulty with flare management, citing slow response to treatment (63%) and lack of flare prevention (66%) as key challenges. There is an urgent need for ...

Null IL36RN mutations are prevalent among East Asian patients with GPP and ACH. Marked ethnic differences in genetic background, clinical presentation, and treatment responses are apparent between ...

The relationship between a parameter readily available on standard electrocardiogram and invasively measured vascular stiffness provides the opportunity to refine patient selection, monitoring, and ...

Pharmacological inhibition of xCT with sulfasalazine, administered preventively or therapeutically, significantly reversed or prevented PAH-related abnormalities. The plasma membrane exchanger xCT may ...

In this study, relative residence time, which is a parameter that takes into account both oscillatory shear stress and time-averaged wall shear stress, emerged as a robust metric for evaluating ...

63% of patients had the V142I variant, which was the only variant among patients diagnosed with a pure neuropathy phenotype. The most common transthyretin (TTR) variant was the V142I variant in a ...

More standardized research is needed to guide second-line agent selection in IgG4-RD. Rituximab offers a highly effective steroid-sparing option for the treatment of immunoglobulin G4-related disease ...