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
<i>Craticula scientiacivica</i> (Stauroneidaceae), sp. nov., a new diatom species found during a citizen science project. [PDF]
Moyón J +4 more
europepmc +1 more source
A Two‐Stage Questionnaire and Actigraphy Screening for iRBD in a Multicenter Retrospective Cohort
ABSTRACT Objective Isolated rapid‐eye‐movement sleep behavior disorder is a prodromal marker of synucleinopathies. However, most cases remain undiagnosed due to the insufficient predictive value of questionnaires and limited access to confirmatory video‐polysomnography. We assessed a two‐stage screening strategy combining a brief questionnaire on rapid‐
Caleb A. Massimi +17 more
wiley +1 more source
Oral Manifestations of Varicella and Their Contribution to Clinical Assessment in Hospitalized and Outpatient Patients. [PDF]
Stoeva V, Kondeva V, Stoyanova R.
europepmc +1 more source
White Matter Microstructural Abnormalities in Neonatal Onset Genetic Epilepsy
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
Digital health technologies in pain management for patients with endometriosis: A scoping review. [PDF]
Wang X, Wang H.
europepmc +1 more source
"If It Don't Talk, It's Not Whakapapa Data": Conceptualising Whakapapa Data in a Digital Age. [PDF]
Pēpi Tarapa-Dewes E, Kukutai T.
europepmc +1 more source
Harmonizing HIV-1 RNA laboratory measurements in a longitudinal cohort collaboration. [PDF]
Lee JS +20 more
europepmc +1 more source

