At the initiative of the Muscular Dystrophy Association (MDA), American experts have reviewed the successes and difficulties encountered with gene therapies mediated by adeno-associated viruses, for all neuromuscular diseases combined:
- this work brought together 47 clinicians and researchers, 41 representatives of the pharmaceutical industry involved in the field and 11 patient representatives,
- an exhaustive review of undesirable effects, particularly immunological, and their mechanisms was carried out, leading to the adoption of practical recommendations,
- the issue of reinjection (theoretical at this stage) of gene therapy products was also addressed.
This group now meets every year and is making progress in overcoming the many obstacles encountered.
