Publications by authors named "Nancy Silliman"
Article Synopsis
- Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a serious, progressive disease, and vutrisiran is a new treatment that works by reducing the production of transthyretin in the liver.
- In a double-blind trial involving 655 patients, those receiving vutrisiran had a lower risk of death and cardiovascular events compared to those on placebo, demonstrating significant efficacy.
- Vutrisiran also improved patient outcomes, showing less decline in walking distance and quality of life measurements over the study period compared to placebo.
Background And Objective: Manifestations of the autosomal recessive disorder lysosomal acid lipase deficiency (LAL-D) include hepatomegaly, elevated serum liver enzymes, and progressive liver disease. We report an analysis of time to progression from first clinical manifestation to first documentation of hepatic fibrosis, cirrhosis, or liver transplantation from an observational study of pediatric and adult patients with LAL-D (clinical trial registration: NCT01528917).
Methods: Data were analyzed from 31 patients with available biopsy data and 1 patient without biopsy data who had undergone liver transplantation.
Objective: To determine the validity, reliability, and feasibility of durometer measurements of skin hardness as an outcome measure in clinical trials of scleroderma.
Methods: Skin hardness was measured during a multicenter treatment trial for scleroderma using handheld digital durometers with a continuous scale. Skin thickness was measured by modified Rodnan skin score (MRSS).
Objective: To evaluate CAT-192, a recombinant human antibody that neutralizes transforming growth factor beta1 (TGFbeta1), in the treatment of early-stage diffuse cutaneous systemic sclerosis (dcSSc).
Methods: Patients with SSc duration of <18 months were randomly assigned to the placebo group or to 1 of 3 CAT-192 treatment groups: 10 mg/kg, 5 mg/kg, 0.5 mg/kg.
Background: Pretreatment antimicrobial resistance has an important impact on the efficacy of many Helicobacter pylori treatment regimens.
Objective: To estimate the prevalence of H. pylori resistance to antimicrobials in the United States, to characterize risk factors associated with H.