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
Pelvic floor complaint-related psychological distress recorded by pelvic physical therapists in the Netherlands: Additional analysis of data from an exploratory file review study. [PDF]
Brand A +3 more
europepmc +1 more source
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
Roper romps, feeling rules, and the "appropriately inappropriate" management of joy. [PDF]
Pearce JS.
europepmc +1 more source
Advancing Stroke Clinical Trials Using Community Engagement and Implementation Science Approaches
ABSTRACT Stroke clinical trials are essential for advancing stroke care but can face challenges with recruitment, retention, clinical relevance, and translation into real‐world practice. We propose that integrating community engagement and implementation science approaches into stroke trials can help address these needs.
Lesli E. Skolarus +2 more
wiley +1 more source
Association between self-reported discrimination and body weight dissatisfaction among adolescents in a city in Santa Catarina, Brazil. [PDF]
Almeida MFDS, Zanlorenci S, Silva DAS.
europepmc +1 more source
Disentangling Climate Worry and Psychological Distress: Data From the UK Household Longitudinal Study. [PDF]
Helminen V, Jokela M, Elovainio M.
europepmc +1 more source
Cognitive Behavioral Therapy for Youth With Childhood‐Onset Lupus: A Randomized Clinical Trial
Objective Our objective was to determine the feasibility and acceptability of the Treatment and Education Approach for Childhood‐Onset Lupus (TEACH), a six‐session cognitive behavioral intervention addressing depressive, fatigue, and pain symptoms, delivered remotely to individual youth with lupus by a trained interventionist.
Natoshia R. Cunningham +29 more
wiley +1 more source
The limited diagnostic value of "feeling a pop" for identifying acute tendon rupture in workers' compensation shoulder claims. [PDF]
Ramtin S, Jayakumar P, Ring D, Tonn M.
europepmc +1 more source
Objective We developed a novel electronic health record sidecar application to visualize key rheumatoid arthritis (RA) outcomes, including disease activity, physical function, and pain, via a patient‐facing graphical interface designed for use during outpatient visits (“RA PRO dashboard”).
Gabriela Schmajuk +16 more
wiley +1 more source

