Results 31 to 40 of about 97,733 (209)
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
Annals of Clinical and Translational Neurology, EarlyView.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
Alexandria Engineering Journal, 2012 Various risk factors influence construction projects cost and schedule performance from project conception to completion. In the context of project management contract assessment helps allocate integrated risks.
Ali F. Bakr +2 more
doaj +1 more source
Bi‐ and Mono‐Allelic RFC1 Expansion in a North American Cohort With Idiopathic Axonal Neuropathy
Annals of Clinical and Translational Neurology, EarlyView.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
Akad Mudharabah dalam Perspektif Fikih dan Perbankan Syariah
Saliha, 2020 This study discusses how the implementation of the mudharabah contract in Islamic banking practices in general. As well as how the position of the mudharabah contract in fiqh and how the mudharabah contract scheme is.
Chasanah Novambar Andiyansari
doaj
Autoimmune Comorbidities as Modifiers of Phenotypic Heterogeneity in Facioscapulohumeral Dystrophy
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Facioscapulohumeral dystrophy type 1 (FSHD1) shows clinical heterogeneity that is only partly explained by D4Z4 repeat unit (RU) size. Although immune and inflammatory mechanisms may contribute to disease variability, the prevalence and clinical impact of autoimmune diseases in FSHD remain unclear.
Jonathan Pini +9 more
wiley +1 more source
Basic Elements of Sponsorship Contract in Sport
Zbornik Radova Pravnog Fakulteta u Splitu, 2018 Sponsorship represents very important source of finances for many sports organizations. The aim of the article is to analyze structural elements of sponsorship contract and to propose a proper definition of a sponsorship contract, while leaning on the ...
Tone Jagodic, Zlatko Mateša
doaj +1 more source
Generalized Dystonia With Cervical Predominance in a Patient With a De Novo TCF20 Variant
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT TCF20 has been implicated in developmental delay and intellectual disability, with dystonia reported less frequently. We describe a woman in her early twenties with childhood‐onset learning difficulties, progressive dysarthria, tremor, and generalized dystonia with marked cervical predominance, associated with a de novo TCF20 frameshift ...
Melisa Kilic +7 more
wiley +1 more source
Invalidity of contract: legislative regulation and types
Проблеми Законності, 2017 Invalidity contracts always attracted attention researchers. Without regard to it, in modern conditions there is an enormous layer of the problems related to invalidity contract, that to require a doctrine and utilitarian comprehension.
Василь Іванович Крат
doaj +1 more source
Discordance Between Patient and Physician Global Assessments in Early Systemic Sclerosis
Arthritis Care &Research, EarlyView.Objective This study aims to identify factors associated with patient global assessment (PtGA) and physician global assessment (PhGA) and discordance between them in systemic sclerosis (SSc). Methods Data from adults with early SSc (<5 years) from the Collaborative National Quality and Efficacy Registry were included.
Ellen Romich +35 more
wiley +1 more source
Immunosuppressive Drug Use in Limited Systemic Sclerosis: An International Survey
Arthritis Care &Research, EarlyView.Objective Current guidelines recommend immunosuppressive treatment for diffuse cutaneous systemic sclerosis but are less clear on their use in limited cutaneous systemic sclerosis (lcSSc) in the absence of internal organ complications. We conducted an international survey to understand current immunosuppressive drug prescribing patterns in lcSSc ...
Sabrina Hoa +3 more
wiley +1 more source