ABSTRACT Objective To clarify the clinical relevance of dopamine transporter single‐photon emission computed tomography (DAT‐SPECT) abnormalities in amyotrophic lateral sclerosis (ALS), with a prespecified focus on sex‐stratified associations with disease progression and short‐term prognosis.
Tomoya Kawazoe +7 more
wiley +1 more source
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
Preservation of the Native Esophagus in Long-Gap Esophageal Atresia: Case Presentation. [PDF]
Suarez Mantilla SN +4 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
The long-term effect of femoral leg lengthening on muscle strength - A 10-year follow-up. [PDF]
Speth BM +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
Obesity Severity Differentially Shapes Diabetes-Related Impairment in Cardiorespiratory Fitness: A Cross-Sectional Propensity Score-Weighted Analysis of Middle-Aged Adults. [PDF]
Jin Y +6 more
europepmc +1 more source
Plastic Deformation of the Pediatric Forearm: Recognition, Radiographic Measurement, and Management. [PDF]
Wetzel C, Rohl M, Manes T, Kadado A.
europepmc +1 more source
Vulvar Aggressive Angiomyxoma in a Postmenopausal Woman: Diagnostic Pitfalls and Surgical Implications-A Case Report. [PDF]
Siferih M, Molla B, Alemneh Y, Desta S.
europepmc +1 more source
Inf-Sup Stable Space-Time Discretization of the Wave Equation Based on a First-Order-In-Time Variational Formulation. [PDF]
Ferrari M, Perugia I, Zampa E.
europepmc +1 more source

