Systemic administration of PRO051 in Duchenne's muscular dystrophy.

By N. Goemans, M. Tulinius, J. T. V. D. van den Akker, B. Burm, P. Ekhart, Niki Heuvelmans, T. Holling, A. Janson, G. Platenburg, J. Sipkens and 9 more. New England Journal of Medicine, 2011

684 citations recorded by the source corpus.

Subfields: Cardiology and Cardiovascular Medicine, Cellular and Molecular Neuroscience, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/220579e350ad8d3d79627c54e32e443fb217569b

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1. J. Mendell
2. J. Gastier-Foster
3. R. al‐Dahhak
4. N. Street
5. N. Leslie
6. K. Flanigan
7. C. Shilling

Citations in the corpus

• Evidence‐based path to newborn screening for duchenne muscular dystrophy (2012, background): “Especially relevant is clinical improvement following the systemic administration of antisense oligonucleotide (AON) PRO051 to induce skipping of exon 51 during premessenger RNA splicing of the DMD gene.(15) Patients enrolled in this exon-skipping trial upregulated dystrophin at the sarcolemma and a”