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Clinical, Histologic, and Pathologic Characteristics of Sporadic Late-onset Nemaline Myopathy

Inverting the Drug Discovery Funnel: In Vivo First Screening for mAb Blood-brain Barrier Penetration Yields Molecules with Novel Uptake Properties

Genetic Work Up in a Consanguineous Family with Variable Phenotypes Including Cognitive Regression, Spasticity, and Visual Impairment