Free Access

Tableau II.

Essais cliniques dans les sarcoglycanopathies.

Pathologie NCT Administration Vecteur Sponsor et  PI Publications
Alpha-SG NCT00494195 Intramusculaire rAAV1.tMCK.human-alpha-sarcoglycan Nationwide Children’s Hospital Mendell JR et al, 2009
Mendell JR et al, 2010
Alpha-SG NCT01976091 Perfusion SRP-9004
scAAVrh74.tMCK.hSGCA
Nationwide Children’s Hospital Mendell JR et al, 2009
Mendell JR et al, 2010
Bêta-SG NCT03652259 Systémique SRP-9003
scAAVrh74.MHCK7.hSGCB
Sarepta Therapeutics, Inc.  
Gamma-SG NCT01344798 Intramusculaire AAV1-des-hgSGC Généthon Herson et al, 2012

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