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 ...
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 ...
This is the first report from SveATTR— a longitudinal Swedish web-based registry open for TTR variant carriers and patients with ATTR amyloidosis. The study summarizes 60 years of data collected as ...
The chronic use of a TGF-β-targeting phosphorodiamidate morpholino oligomer (PMO) and dystrophin exon-skipping PMO reduced muscle fibrosis and loss, restored dystrophin expression in skeletal and ...
The average telomere loss in patients with SSc was approximately 223 base pairs per year, compared with 30-60 pairs per year in normal aging. Shortened peripheral blood leukocyte telomere length ...
Feature importance analysis using Shapley additive explanations values identified right atrial (RA) and PA volumes as the most influential features for the new model. A novel, fully automated random ...
After 26 weeks, children with achondroplasia who were treatment-naive achieved an increase in annualized growth velocity from 4.92 to 9.14 cm/year, whereas children previously treated with ...
RN3271 showed deep and durable effects in reducing TTR protein with a favorable safety profile. MINNEAPOLIS— RN3271, a novel GalNAc-conjugated siRNA designed to silence transthyretin (TTR) expression ...
Seventeen of the 22 ultimately diagnosed with cMCD had relatively low basal serum tryptase, and 64% initially tested negative for peripheral blood KIT p.D816V. SM is a rare disorder involving the ...
The majority (90%) of neurologists were aware of 40 out of 61 recommendations on the treatment of myasthenia gravis (MG). Most Spanish neurologists know, agree on, and apply guidelines for the ...
Transthyretin amyloids-specific drugs led to a trend toward less decline in the 6-minute walk distance and were associated with a significantly blunted decline in Kansas-City ...
Patients who live with rare conditions have specialty care teams. You have a primary doctor, and you have other doctors who specialize in the care you need based on your condition. I have Pompe ...
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