Results 131 to 140 of about 5,675,213 (309)
Clinical profile of invasive fungal CNS infections in children with ALL a single center study
Pediatric Hematology Oncology Journal, 2022 Dhara Shah +10 more
doaj +1 more source
Effectiveness and safety of Risdiplam for types 1–3 spinal muscular atrophy in a single center
BMC NeurologyBackground Risdiplam has demonstrated efficacy in various types of 5q-spinal muscular atrophy (SMA) during clinical trials, yet real-world data remain limited.
Xiaomei Zhu +6 more
doaj +1 more source
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit +11 more
wiley +1 more source
Children\u27s Mercy Hospital Annual Report 2014
, 2015 Annual report for The Children\u27s Mercy Hospital in Kansas City MO, a pediatric medical center.https://scholarlyexchange.childrensmercy.org/cmh-annual-reports/1008/thumbnail ...
Children\u27s Mercy Hospital
core
For All Children Everywhere: Community Benefit Report 2016
, 2017 Fiscal year report on the community benefit provided by Children\u27s Mercy Kansas City to the communities in the Kansas City ...
Children\u27s Mercy Hospital
core +1 more source
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
White Matter Microstructural Abnormalities in Neonatal Onset Genetic Epilepsy
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Recent evidence indicates that epilepsy is associated with abnormal white matter. If seizures alter white matter, then the impact upon network function, epileptogenesis, and cognition could be pronounced in neonates undergoing rapid developmental myelination. Neonates with epilepsy due to nonstructural genetic causes provide a unique
Amanda G. Sandoval Karamian +8 more
wiley +1 more source
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Down syndrome regression disorder is a syndrome characterized by subacute loss of cognitive, behavioral, and functional abilities in individuals with Down syndrome. Electroencephalography abnormalities are frequently observed during evaluation, but it remains unclear whether these findings represent a dynamic marker of disease ...
Jonathan D. Santoro +14 more
wiley +1 more source
Children\u27s Mercy Annual Report 2004
, 2005 Annual report for The Children\u27s Mercy Hospital in Kansas City MO, a pediatric medical center.https://scholarlyexchange.childrensmercy.org/cmh-annual-reports/1013/thumbnail ...
Children\u27s Mercy Hospital
core
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objectives Focal cortical dysplasia (FCD) is the most common etiology of drug‐resistant epilepsy in children. Focal to bilateral tonic–clonic seizures (FBTCS) mark a high risk of drug‐resistant epilepsy and involve thalamocortical circuitry in their generation and propagation.
Hua Xie +8 more
wiley +1 more source