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
Deconfounded-Debiased Estimation and Inference for High-Dimensional Mediation Analysis With Pervasive Hidden Confounders. [PDF]
Stat MedLi Z, Pan L, Yu Y, Qin G, Fu B.
europepmc +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
Asymptotic Normality and Convergence Rates for Tsallis Entropy Estimators via Stabilization Techniques. [PDF]
Entropy (Basel)Çadırcı MS, Singull M.
europepmc +1 more source
A Two‐Stage Questionnaire and Actigraphy Screening for iRBD in a Multicenter Retrospective Cohort
Annals of Clinical and Translational Neurology, EarlyView.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
Conditional Estimations for Seamless Phase II/III Clinical Trials Involving Multi-Stage Early Stopping. [PDF]
Stat MedZhu S +6 more
europepmc +1 more source
Transformation-based median estimation under skewed-symmetric distributions with long-memory data applications. [PDF]
Sci RepDaraz U, Aljohani HM, Alshanbari HM.
europepmc +1 more source
Estimating the Growth Rate of a Birth and Death Process Using data From a Small Sample. [PDF]
Bull Math BiolHeinzel CS, Schweinsberg J.
europepmc +1 more source
HIGH-DIMENSIONAL NEWEY-POWELL TEST VIA APPROXIMATE MESSAGE PASSING. [PDF]
Econ Theory (N Y)Zhou J, Zou H.
europepmc +1 more source