Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders.

Finkel, Richard (author)
Wong, Brenda (author)
Barohn, Richard (author)
Campbell, Craig (author)
Comi, Giacomo P (author)
Connolly, Anne M (author)
Day, John W (author)
Flanigan, Kevin M (author)
Goemans, Nathalie (author)
Jones, Kristi J (author)
Mercuri, Eugenio (author)
Quinlivan, Ros (author)
Renfroe, James B (author)
Russman, Barry (author)
Ryan, Monique M (author)
Tulinius, Mar,1953Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xtulim (author)
Voit, Thomas (author)
Moore, Steven A (author)
Lee Sweeney, H (author)
Abresch, Richard T (author)
Coleman, Kim L (author)
Eagle, Michelle (author)
Florence, Julaine (author)
Gappmaier, Eduard (author)
Glanzman, Allan M (author)
Henricson, Erik (author)
Barth, Jay (author)
Elfring, Gary L (author)
Reha, Allen (author)
Spiegel, Robert J (author)
O'donnell, Michael W (author)
Peltz, Stuart W (author)
McDonald, Craig M (author)
Göteborgs universitetInstitutionen för kliniska vetenskaper, Avdelningen för pediatrik (creator_code:org_t)

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MEDICAL AND HEALTH SCIENCES: MEDICAL AND HEAL ...; and Clinical Medicin ...; and Pediatrics

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