Key outcomes included high ILD prevalence, postmyopathic PPF in 13.5% of the cohort, higher rates of rapidly progressive ILD and transplantation in postmyopathic PPF, greater rituximab use in PPF, and ...
Racial and ethnic inequities persist in MG severity and outcomes, with a particularly high disease burden among Indigenous patients. Rare Disease Advisor, a trusted source of medical news and feature ...
Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...
Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida in Gainesville who specializes in ...
Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...
Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...
Castleman disease and IgG4-RD have overlapping disease manifestations that can complicate the diagnostic process. A male patient initially suspected of having immunoglobulin G4-related disease ...
The recommendations were based on the available evidence, its certainty, and key judgments using the Grading of Recommendations Assessment, Development and Evaluation framework. New guidelines ...
The modified Spinal Muscular Atrophy Functional Rating Scale (SMAFRS) is a psychometrically stable and unidimensional outcome measure, even though certain items are misfitting like those related to ...
From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...
In a nonhuman primate model of acquired hemophilia A, treatment with PS-siRNA 40031 restored thrombin generation to normal levels. Reducing protein S (PS) levels can increase thrombin generation and ...
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