Our Pipeline
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Duchenne Muscular Dystrophy Exon Skipping
(Phase 2/3 Clinical Trial)
%
Duchenne Muscular Dystrophy
(Gene Therapy Preclinical Studies)
%
Spinal Muscular Atrophy Antisense
(Ready for Clinical Trial)
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Ataxia telangiectasia Antisense
(In vitro studies)