Results 261 to 270 of about 5,330,848 (365)

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

Evaluating Large Language Models for Patient Information: What Is Worth Publishing? [PDF]

open access: yesFoot Ankle Orthop
Saltzman CL   +9 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

Practice Recommendations for Genetic Testing of Ataxias

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Over the past decade, significant advances in genetic testing for ataxia have improved diagnostic accuracy, informed clinical trial eligibility, guided treatment decisions, and enabled cascade testing of at‐risk relatives. While guidance exists for other neurogenetic conditions, there are no standardized guidelines on genetic ...
Sharan R. Srinivasan   +7 more
wiley   +1 more source

Gastrointestinal Perforation as a Safety Concern Among Patients With Rheumatoid Arthritis Receiving JAK Inhibitor Therapy: A Systematic Review and Network Meta‐Analysis

open access: yesArthritis Care &Research, EarlyView.
Objective Gastrointestinal perforation (GIP) is a rare and life‐threatening safety concern associated with JAK inhibitors (JAKi). We aimed to review the evidence regarding the risk of GIP associated with the use of JAKi in patients with rheumatoid arthritis (RA) using a systematic review and network meta‐analysis approach.
Thipsukhon Sathapanasiri   +7 more
wiley   +1 more source

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