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WHO 11362
H4T32JIH01
PF-06939926
FORDADISTROGEN MOVAPARVOVEC
A REPLICATION-DEFECTIVE ADENO-ASSOCIATED VIRAL VECTOR ENCODING A CODON-OPTIMISED MINI-DYSTROPHIN (DMD). A RECOMBINANT REPLICATION-DEFECTIVE ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 9 (RAAV9), ENCODING A CODONOPTIMISED MINI-DYSTROPHIN (DMD) GENE COMPRISING THE N-TERMINUS, HINGE REGIONS H1, H3 AND H4, SPECTRIN-LIKE REPEATS R1, R2 AND R22-24, AND THE C-TERMINAL CYSTEINE-RICH REGION, UNDER THE CONTROL OF A HUMAN MUSCLE-SPECIFIC SYNTHETIC ENHANCER/PROMOTER AND A SYNTHETIC POLY(A) SEQUENCE, FLANKED BY AAV2 INVERTED TERMINAL REPEATS (ITR)