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Q35069526-2FBFF6AD-B8E6-4C61-9021-DF475294FAC7
Q35069526-2FBFF6AD-B8E6-4C61-9021-DF475294FAC7
BestRank
Statement
http://www.wikidata.org/entity/statement/Q35069526-2FBFF6AD-B8E6-4C61-9021-DF475294FAC7
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.
P2860
Q35069526-2FBFF6AD-B8E6-4C61-9021-DF475294FAC7
BestRank
Statement
http://www.wikidata.org/entity/statement/Q35069526-2FBFF6AD-B8E6-4C61-9021-DF475294FAC7
rank
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wasDerivedFrom
33cd1a38b0c1c142bae634a9ca607a17db954f8e
P2860
Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.