American and European clinicians involved in innovative therapies for Duchenne muscular dystrophy (DMD) have published guidelines for the use of delandistrogene moxeparvovec, a gene therapy product:
- these recommendations are the result of experience acquired during the clinical development of this innovative molecule, which includes an AAV vector of the rh74 type and was developed by the Sarepta laboratory,
- they include various biological tests prior to a single injection of this product, including the level of antibodies against Rh74-type AAV,
- the oral administration of corticosteroids to prevent any risk of immune reaction is described in detail, as are short- and medium-term clinical and biological monitoring procedures for children receiving such treatment.
