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- W2007923635 abstract "Duchenne Muscular Dystrophy is due to the absence of dystrophin in the sarcolemma. We have done a phase 1 clinical trial conducted on DMD patients aged between 5 and 17 year old. Allogenic myoblasts were transplantation in 1 cm3 of muscle and patients were immunosuppressed for 1 month with tacrolimus. This restored the expression of dystrophin in up to 34% of muscle fibers. We are currently trying to do a Phase 2 trial to verify whether the transplantation of myoblasts throughout a muscle can increase its strength. The patients would have to be immunosuppressed during 6 months with tacrolimus. However, due to the perceived risks associated with immunosuppression, the Human Ethic Committee of our hospital and Health Canada have restricted participation to DMD patients who are more than 18 years old, are able to move the wrist on both side (for strength measurements), are not on a respirator and are not seropositive for CMV. As DMD is a rare disease, these restrictions make recruitment of 10 patients impossible in Canada. Moreover, DMD patients more than 18 years old are at a very advanced stage of the disease and their muscles have been largely substituted by fat and conjunctive tissue. Thus the transplanted myoblasts are less likely to be able to fuse with existing patient muscle fibers to introduce nuclei coding for the normal dystrophin and to increase the size of these fibers. Thus these older DMD patients are not the best candidates to verify the beneficial effects of myoblast transplantation. The restriction in the participation of minors is due to the perceived risks of tacrolimus, yet this drug is not only used for organ transplantation in children but is also under clinical trial for type I diabetes in children, a disease for which there is some treatment and which is much less severe than DMD. Thus support of the international scientific community is required to convince the regulatory agencies to revise their restrictions." @default.
- W2007923635 created "2016-06-24" @default.
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- W2007923635 date "2009-09-01" @default.
- W2007923635 modified "2023-10-17" @default.
- W2007923635 title "T.P.6.09 Will it be possible to do a phase 2 clinical trial of myoblast transplantation for DMD patients?" @default.
- W2007923635 doi "https://doi.org/10.1016/j.nmd.2009.06.284" @default.
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