DMD

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anti-inflammation

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Treat symptoms not cure

NF-kB inhibitors

corticosteroids

edasalonexent (Ph3)

vamorolone (Ph2)

Emflaza (M)

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Target Dystrophin

Partially restore the function or expression of dystrophin

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microdystrophin (learn from BMD)

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complement activation concerned

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PF-06939926 (Ph1)

SRP-9001 (Ph2/3)

SGT-001 (clinical hold)

Exon skipping

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PMO (pre-mRNA)

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Exon 51 (13%)

Exon 53 (8%)

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PPMO: improve muscle cell expression

golodirsen (M)

eteplrsen (M)

SRP-5051 (Ph2)

NS-065 (NDA)

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