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#661B62
87
EXONDYS 51 (eteplirsen)*
#661B62
87
VYONDYS 53 (golodirsen)*
#661B62
65
Casimersen (SRP-4045)
#661B62
40
Exon 52
#661B62
20
Exon 43
#661B62
20
Exon 44
#661B62
20
Exon 50
#661B62
20
Exon 55
#661B62
65
SRP-5051
#661B62
40
SRP-5053
#661B62
40
SRP-5045
#661B62
40
SRP-5052
#661B62
40
SRP-5044
#661B62
40
SRP-5050
#661B62
65
SRP-9001 Micro-dystrophin
#661B62
65
GALGT2 (Nationwide Children's)
#661B62
40
Micro-dystrophin (Genethon)
#3453A3
65
SRP-9003 (LGMD2E β-sarcoglycan)
#3453A3
65
SRP-9004 (LGMD2D α-sarcoglycan)
#3453A3
40
SRP-9005 (LGMD2C γ-sarcoglycan)
#3453A3
65
SRP-6004 (LGMD2B Dysferlin)
#3453A3
40
SRP-9006 (LGMD2L Anoctamin 5)
#3453A3
40
Calpain 3 (LGMD2A) (Nationwide Children's)
#15587B
40
Neurotrophin 3 (CMT 1A) (Nationwide Children's)
#8694A4
65
LYS-SAF302 (MPS IIIA) (Lysogene)
#8EAA6D
40
Cardiomyopathy (University of Florida)
#999899
20
Pompe Disease (Lacerta)
#999899
20
CNS-1 (Lacerta)
#999899
20
Multiple Sclerosis (University of Florida)
#999899
20
Rett Syndrome (University of Massachusetts)
#999899
20
Rett Syndrome (StrideBio)
#999899
20
Dravet Syndrome (StrideBio)
#999899
20
Angelman Syndrome (StrideBio)
#999899
20
Niemann-Pick Type C (StrideBio)
#999899
20
Muscle/CNS Target 1 (StrideBio)
#999899
20
Muscle/CNS Target 2 (StrideBio)
#999899
20
Muscle/CNS Target 3 (StrideBio)
#999899
20
Muscle/CNS Target 4 (StrideBio)
#999899
20
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University)
#661B62
20
Duchenne (Institute of Myology)**
#661B62
20
CRISPR/CAS9 (Duke University)
#661B62
20
Duchenne (Harvard University)

* Candidate received accelerated approval in the U.S., confirmatory studies required
** Gene therapy and PPMO

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