Results 271 to 280 of about 2,417,970 (339)

Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
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

SYHA1813, A VEGFR and CSF1R Inhibitor, in Patients With Recurrent High‐Grade Gliomas: A Multicenter, Open‐Label Phase I Study

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
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

The contribution of minimally invasive tissue sampling compared to antemortem-derived cause of death determination among inpatient child deaths: the minimally invasive tissue sampling in Malawi study. [PDF]

open access: yesJ Glob Health
Voskuijl WP   +18 more
europepmc   +1 more source

Durability of Response to B‐Cell Maturation Antigen‐Directed mRNA Cell Therapy in Myasthenia Gravis

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective We report the 12‐month follow‐up outcomes from a Phase 2 clinical trial (NCT04146051) evaluating Descartes‐08, a BCMA‐directed RNA chimeric antigen receptor T‐cell (rCAR‐T) therapy for refractory generalized myasthenia gravis (MG).
Nizar Chahin   +10 more
wiley   +1 more source

Metabolic Consequences of Rheumatoid Arthritis

open access: yesArthritis Care &Research, EarlyView.
Patients with rheumatoid arthritis (RA) may have metabolic disruption, which can contribute to adverse long‐term outcomes, for multiple reasons. Patients with RA appear to have a higher risk of sarcopenia, type 1 and type 2 diabetes mellitus, metabolic syndrome, and hypertension. Systemic inflammation in RA can cause a “lipid paradox,” with reduced low‐
Stevie Barry   +2 more
wiley   +1 more source

Real‐World Cost‐Effectiveness of a Standardized Education and Exercise Therapy Program for Hip and Knee Osteoarthritis Compared to Usual Care

open access: yesArthritis Care &Research, EarlyView.
Objective We estimated the real‐world cost‐effectiveness of a standardized education and exercise therapy program (GLA:D) compared to usual care (UC) for people managing hip and/or knee osteoarthritis (HKOA). Methods We used a prospective matched cohort design to recruit people (aged >45 years) diagnosed with HKOA who used GLA:D or UC (not on a ...
Darren R. Mazzei   +4 more
wiley   +1 more source

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