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
Introduction of Internal Accounting Control System & Conservatism of Accounting Information
전흥주, 박정대
openalex +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
Transcriptome characterisation and population genetics of <i>Cunninghamiakonishii</i> Hayata - An endangered gymnosperm and implication for its conservation in Vietnam. [PDF]
Ha BH+6 more
europepmc +1 more source
Durability of Response to B‐Cell Maturation Antigen‐Directed mRNA Cell Therapy in Myasthenia Gravis
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
Developing a carbon footprint calculation method for product life cycle based on low-carbon design: A case study of the STAGE Bluetooth speaker. [PDF]
Zhang Y, Yao S, Chen Y.
europepmc +1 more source
Practice Recommendations for Genetic Testing of Ataxias
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
Reconceptualizing the balanced scorecard as a communication mechanism in healthcare. [PDF]
Guo F, Huang YS, Nemoto M.
europepmc +1 more source