Results 171 to 180 of about 1,997,596 (295)
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
Children and Wealth Contexts in the United States: Differences by Household Type. [PDF]
Gibson-Davis C.
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
Brief admission by self-referral for adolescents who self-harm: discourses on involvement and responsibility among parents and other significant adults. [PDF]
Lantto R+5 more
europepmc +1 more source
Genetic Modifiers of Parkinson's Disease: A Case–Control Study
ABSTRACT Objective To examine the associations of LRRK2 p.G2019S, GBA1 p.N409S, polygenic risk scores (PRS), and APOE E4 on PD penetrance, risk, and symptoms. Methods We conducted a US‐based observational case–control study using data from the 23andMe Inc. and Fox Insight Genetic Substudy (FIGS) databases.
Matthew J. Kmiecik+15 more
wiley +1 more source
Understanding the relationship between child stimulation and brain function using neuroimaging techniques and behavioral measures: a study protocol. [PDF]
Kitsao-Wekulo P+5 more
europepmc +1 more source