Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
Hydrolyzed Rice Formula for the Management of Cow's Milk Allergy in Infants and Young Children: A Systematic Review of Randomized Controlled Trials. [PDF]
Szajewska H +7 more
europepmc +1 more source
ABSTRACT Background Ischemic stroke, a major cause of mortality and long‐term disability, results from the abrupt cessation of cerebral blood flow due to vascular occlusion or rupture. Icosapent Ethyl (EPA‐EE), approved for hypertriglyceridemia, has anti‐inflammatory and antithrombotic properties that may lessen ischemic damage.
Mitra Mahmoudi Meymand +5 more
wiley +1 more source
DEPREDATION CLAIM BEHAVIOR AND TOLERANCE OF WILDLIFE IN WYOMING [PDF]
Wyoming Game and Fish Department depredation payments were established to increase landowner tolerance toward, and this the supply of, certain types of wildlife.
Van Tassell, Larry W. +2 more
core
Limitations in the Assessment of Pain Reporting Reliability-Validation of the Focused Analgesia Selection Test. [PDF]
Milde C +3 more
europepmc +1 more source
Bi‐ and Mono‐Allelic RFC1 Expansion in a North American Cohort With Idiopathic Axonal Neuropathy
ABSTRACT Objective RFC1 biallelic repeat expansion is increasingly recognized as a cause of chronic idiopathic axonal polyneuropathy (CIAP), but it remains challenging to know who to test. This study aims to determine the prevalence of biallelic and monoallelic RFC1 expansions and their corresponding neuropathy phenotypes in CIAP patients and identify ...
Amro M. Stino +25 more
wiley +1 more source
Low Frustration Tolerance, Childhood Perfectionism, and Mental Health Problems in Peruvian Children: A Network Analysis. [PDF]
Angulo-Salas R +2 more
europepmc +1 more source
ABSTRACT Objective Treatment of disorders of consciousness (DoC) remains a major clinical challenge, and noninvasive, targeted modulation of deep brain structures has emerged as a promising therapeutic strategy. We aimed to evaluate the feasibility/safety and preliminary effects of thalamic temporal interference stimulation (TIS) targeting centromedian‐
Gengyao Hu +7 more
wiley +1 more source
Promoting tolerance to schools in a time of extremism
Tolerance is arguably both morally ambiguous and politically essential. School leaders in England have an obligation and a legal requirement: to encourage acceptance of difference and diversity, declare in public terms, that which is unacceptable and ...
Bowie, R. A.
core
Regulatory Mechanisms of BBX Transcription Factors in Plant Responses to Abiotic Stress and Light Signals. [PDF]
Chen P, Meng J, Kong D, Cao J, Han R.
europepmc +1 more source

