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DMD (anti-inflammation (NF-kB inhibitors (edasalonexent (Ph3), vamorolone…
DMD
anti-inflammation
NF-kB inhibitors
edasalonexent (Ph3)
vamorolone (Ph2)
corticosteroids
Emflaza (M)
Target Dystrophin
PMO (pre-mRNA)
Exon 51 (13%)
PPMO: improve muscle cell expression
SRP-5051 (Ph2)
eteplrsen (M)
Exon 53 (8%)
golodirsen (M)
NS-065 (NDA)
microdystrophin (learn from BMD)
SRP-9001 (Ph2/3)
complement activation concerned
SGT-001 (clinical hold)
PF-06939926 (Ph1)
Treat symptoms not cure
Partially restore the function or expression of dystrophin
Exon skipping