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
<i>Verticillium</i>-like Anamorphic Fungi in Sordariomycetes from Southwestern China: Two New Taxa and a New Record. [PDF]
Dong QY +5 more
europepmc +1 more source
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
Morphological Diversity and Preliminary DNA Barcoding of <i>Xylaria</i> (Xylariales) from Estación Científica San Francisco, Including <i>Xylaria aenea</i> as a New Record for Ecuador. [PDF]
Cruz D +5 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
New record of Gracilariaphuquocensis (Gracilariaceae, Rhodophyta) in the Philippines. [PDF]
Dumilag RV +4 more
europepmc +1 more source
ABSTRACT Objectives We aimed to determine the frequency of subclinical optic nerve (ON) lesions using MRI, optical coherence tomography (OCT), and visual evoked potentials (VEP) in radiologically isolated syndrome (RIS), and to assess their diagnostic and prognostic significance.
Christine Lebrun‐Frenay +13 more
wiley +1 more source
Three new species and a new record of the genus <i>Lipolexis</i> (Hymenoptera, Braconidae, Aphidiinae) from South Korea. [PDF]
Kim S, Sohn J, Kim H.
europepmc +1 more source
Four new species and a new record of <i>Synagelides</i> Strand, 1906 (Araneae, Salticidae) from China. [PDF]
Liu BL +6 more
europepmc +1 more source
Three novel species and a new record of Pleosporales (Didymellaceae, Roussoellaceae) from China. [PDF]
Guo SQ +8 more
europepmc +1 more source

