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Eteplirsen for the treatment of Duchenne muscular dystrophy.

Annals of neurology | 2013

In prior open-label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic mutations amenable to skipping exon 51. The present study used a double-blind placebo-controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6-minute walk test (6MWT).

Pubmed ID: 23907995 RIS Download

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Associated grants

  • Agency: NCRR NIH HHS, United States
    Id: UL1 RR024992

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BIOQUANT Life Science (software resource)

RRID:SCR_016423

Software tool to collect and analyse data on images of tissue slides or welled plates.Supports high-throughput immunofluorescence and immunohistochemistry, stereology, densitometry, and 3D modeling. Used in bioscience research in animal models and human biopsy, developmental neuroscience, traumatic brain/spinal cord injury, glaucoma, eye-movement disorders, cardiovascular disease and muscle disorders.

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