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Q33807788-47ABA584-2FBF-4B86-BF3D-4DDBD3984E71
Q33807788-47ABA584-2FBF-4B86-BF3D-4DDBD3984E71
BestRank
Statement
http://www.wikidata.org/entity/statement/Q33807788-47ABA584-2FBF-4B86-BF3D-4DDBD3984E71
LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts
P2860
Q33807788-47ABA584-2FBF-4B86-BF3D-4DDBD3984E71
BestRank
Statement
http://www.wikidata.org/entity/statement/Q33807788-47ABA584-2FBF-4B86-BF3D-4DDBD3984E71
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type
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wasDerivedFrom
7f6a842d10e378fe00fad986f012c3e31695dadd
P2860
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.