Building Real-World Evidence Infrastructure to Improve Health and Healthcare in the United States: Part III-Questions for Translational Science and Action Theory. [PDF]
Yogurtcu ON +13 more
europepmc +1 more source
Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice
ABSTRACT Objective The aim of our study was to establish the prevalence of adverse events in a real‐world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. Methods The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean ...
Marika Pane +19 more
wiley +1 more source
Building on the Translational Science Benefits Model to include team science: a practical and theory-based approach to continuous quality improvement and impact evaluation for Clinical and Translational Science Award programs. [PDF]
Brimhall KC +3 more
europepmc +1 more source
Topic analysis on publications and patents toward fully automated translational science benefits model impact extraction. [PDF]
Manjunath T +5 more
europepmc +1 more source
Movement Disorders in Aicardi–Goutières Syndrome and Response to Immunomodulation
ABSTRACT This study characterizes movement disorders and treatment responses in seven children with Aicardi–Goutières syndrome (AGS). We retrospectively evaluated motor phenotypes, neuroimaging, and interferon signatures in patients treated with baricitinib or anifrolumab. Spasticity affected all patients, while dystonia was present in 4/7.
Enrique Gonzalez Saez‐Diez +10 more
wiley +1 more source
Who benefits? Health equity and the Translational Science Benefits Model. [PDF]
La Manna A +10 more
europepmc +1 more source
Annals of Clinical and Translational Neurology, EarlyView.
Qichao Fang, Luting Ying, Yongjin Gong
wiley +1 more source
Impact of Plasma p‐tau181 on Cognition, Motor Phenotypes, and Disease Course in ALS
ABSTRACT Phosphorylated tau181 (p‐tau181), an Alzheimer's disease biomarker, was recently evaluated in amyotrophic lateral sclerosis (ALS). We investigated plasma p‐tau181 in 202 ALS/ALS‐FTD patients and 94 healthy controls, assessing cognitive performance, motor function, and longitudinal dynamics.
Elisabeth Kasper +25 more
wiley +1 more source
Objective We aimed to validate the Pediatric Arthritis Ultrasound Scoring System (PAUSS) for upper extremity joints in children with juvenile idiopathic arthritis (JIA). Methods Children with JIA were evaluated for elbow, wrist, or finger arthritis by clinical examination (CE) and musculoskeletal ultrasound (MSUS) with images scored according to the ...
Patricia Vega‐Fernandez +12 more
wiley +1 more source
Objective Mycophenolate mofetil (MMF) use in limited cutaneous systemic sclerosis (lcSSc) is relatively uncommon because of the lower fibrotic burden and the predominance of vascular complications. In vitro observations and clinical data from transplanted patients suggest a protective effect of MMF on endothelial function.
Enrico De Lorenzis +77 more
wiley +1 more source

