Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons
ABSTRACT Objectives This ongoing, open‐label extension study is evaluating the long‐term safety, tolerability, and efficacy of givinostat, a Class I and II histone deacetylase inhibitor, in patients with Duchenne muscular dystrophy (DMD). Methods The recruited patients completed one of two prior clinical studies (one Phase 2 and one Phase 3 [EPIDYS ...
Craig M. McDonald+74 more
wiley +1 more source
Expansion microscopy reveals nano-scale insights into the human neuromuscular junction. [PDF]
Ramadan A+6 more
europepmc +1 more source
ABSTRACT Objective Recurrent high‐grade gliomas have a poor prognosis and limited therapeutic options. This study aimed to evaluate the safety and efficacy of SYHA1813, a dual inhibitor of VEGFR and CSF1R, in patients with recurrent high‐grade gliomas.
Zhuang Kang+16 more
wiley +1 more source
Polyacrylic acid-polyvinylpyrrolidone complex for achieving hemostasis after hemodialysis: study protocol for an open-label crossover randomized controlled trial (PAA-PVP study). [PDF]
Terashima R+13 more
europepmc +1 more source
CSF Mitochondrial DNA: Biomarker of Body Composition and Energy Metabolism in Parkinson's Disease
ABSTRACT Objective Cerebrospinal fluid (CSF) cell‐free mitochondrial DNA (cf‐mtDNA) is a potential biomarker for Parkinson's disease (PD), but its clinical relevance remains unclear. We investigated associations between CSF cf‐mtDNA levels, body composition, nutritional status, and metabolic biomarkers in PD. Methods CSF cf‐mtDNA levels, defined as the
Yasuaki Mizutani+11 more
wiley +1 more source
Objective We aimed to assess whether high‐sensitivity C‐reactive protein (hsCRP) levels could predict the development of psoriatic arthritis (PsA) in patients with psoriasis. Methods We analyzed data from a prospective cohort of patients with psoriasis without PsA at enrollment.
Lihi Eder+5 more
wiley +1 more source
Assessment of a Stepwise Intervention to Improve Nurse-administered Penicillin Allergy Screening and De-labeling in Pediatric Inpatients. [PDF]
Konold VJL+10 more
europepmc +1 more source
Objective Social determinants of health (SDoH) can impact outcomes but are not routinely screened for in US outpatient rheumatology clinics. This study determined the feasibility of routine point‐of‐care SDoH screening among patients with systemic lupus erythematosus (SLE) and associated barriers and facilitators at the physician, care team, and ...
S. Sam Lim+11 more
wiley +1 more source
Anxiolysis for laceration repair in children: statistical analysis plan for an open-label multicenter adaptive trial (ALICE). [PDF]
Jiang A+3 more
europepmc +1 more source
Objectives Reports have linked both high and low serum uric acid (SUA) levels to adverse health outcomes. This study aimed to establish a reference interval for SUA in older adults and assessed its association with clinically relevant outcomes in relatively healthy, community‐dwelling individuals aged 70 and older.
Amanda J Rickard+15 more
wiley +1 more source