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Cell-mediated exon 51 skipping using autologous mesoangioblasts in Duchenne muscular dystrophy
2024-514860-14-00Phase 2Authorised
- Sponsor
- IRCCS Ospedale San Raffaele
- Start
- Oct 2024
- Modality
- Monoclonal Antibodies → Antibody-Based Therapeutics
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of DMD06-MAB in Duchenne muscular dystrophy (DMD), sponsored by IRCCS Ospedale San Raffaele. Primary endpoints: Safety of DMD06-MAB intramuscular injection and Restoration of dystrophin expression in transplanted muscle. Planned start Oct 2024; status: authorised.
02
Drugs
DMD06-MAB
03
Conditions
Duchenne muscular dystrophy (DMD)
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