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1
Eteplirsen for the treatment of Duchenne muscular dystrophy
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Artigo
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Eteplirsen for the treatment of Duchenne muscular dystrophy

Mendell, Jerry R. ; Rodino-Klapac, Louise R. ; Sahenk, Zarife ; Roush, Kandice ; Bird, Loren ; Lowes, Linda P. ; Alfano, Lindsay ; Gomez, Ann Maria ; Lewis, Sarah ; Kota, Janaiah ; Malik, Vinod ; Shontz, Kim ; Walker, Christopher M. ; Flanigan, Kevin M. ; Corridore, Marco ; Kean, John R. ; Allen, Hugh D. ; Shilling, Chris ; Melia, Kathleen R. ; Sazani, Peter ; Saoud, Jay B. ; Kaye, Edward M.

Annals of neurology, 2013-11, Vol.74 (5), p.637-647 [Periódico revisado por pares]

United States: Blackwell Publishing Ltd

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2
Evidence-based path to newborn screening for duchenne muscular dystrophy
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Artigo
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Evidence-based path to newborn screening for duchenne muscular dystrophy

Mendell, Jerry R. ; Shilling, Chris ; Leslie, Nancy D. ; Flanigan, Kevin M. ; al-Dahhak, Roula ; Gastier-Foster, Julie ; Kneile, Kelley ; Dunn, Diane M. ; Duval, Brett ; Aoyagi, Alexander ; Hamil, Cindy ; Mahmoud, Maha ; Roush, Kandice ; Bird, Lauren ; Rankin, Chelsea ; Lilly, Heather ; Street, Natalie ; Chandrasekar, Ram ; Weiss, Robert B.

Annals of neurology, 2012-03, Vol.71 (3), p.304-313 [Periódico revisado por pares]

Hoboken: Wiley Subscription Services, Inc., A Wiley Company

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3
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
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Artigo
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Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

Mendell, Jerry R. ; Rodino-Klapac, Louise R. ; Rosales, Xiomara Q. ; Coley, Brian D. ; Galloway, Gloria ; Lewis, Sarah ; Malik, Vinod ; Shilling, Chris ; Byrne, Barry J. ; Conlon, Thomas ; Campbell, Katherine J. ; Bremer, William G. ; Taylor, Laura E. ; Flanigan, Kevin M. ; Gastier-Foster, Julie M. ; Astbury, Caroline ; Kota, Janaiah ; Sahenk, Zarife ; Walker, Christopher M. ; Clark, K. Reed

Annals of neurology, 2010-11, Vol.68 (5), p.629-638 [Periódico revisado por pares]

Hoboken: Wiley Subscription Services, Inc., A Wiley Company

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4
Sustained alpha-sarcoglycan gene expression following gene transfer in LGMD2D
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Artigo
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Sustained alpha-sarcoglycan gene expression following gene transfer in LGMD2D

Mendell, Jerry R. ; Rodino-Klapac, Louise R. ; Rosales, Xiomara Q. ; Coley, Brian D. ; Galloway, Gloria ; Lewis, Sarah ; Malik, Vinod ; Shilling, Chris ; Byrne, Barry J. ; Conlon, Thomas ; Campbell, Katherine J. ; Bremer, William G. ; Taylor, Laura E. ; Flanigan, Kevin M. ; Kota, Janaiah ; Sahenk, Zarife ; Walker, Christopher M. ; Clark, K. Reed

Annals of neurology, 2010-11, Vol.68 (5), p.629-638 [Periódico revisado por pares]

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