Results 251 to 260 of about 673,880 (301)

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane   +43 more
wiley   +1 more source

Addressing laboratory challenges in clinical trials: approaches from the Clinical Diagnostic Laboratory Services at MRC/UVRI and LSHTM Uganda Research Unit. [PDF]

open access: yesTrials
Ayebazibwe GK   +11 more
europepmc   +1 more source

Beyond numbers: the missing conceptual foundation in evaluating Mexico's health system performance. [PDF]

open access: yesLancet Reg Health Am
Arreola-Ornelas H   +4 more
europepmc   +1 more source

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