Biological preparations for medical use in the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Pharmaceutical preparations, namely, recombinant adeno-associated viral (rAAV) vectors for use in gene therapy; Pharmaceutical preparations for the treatment of genetic diseases and disorders, and muscle disorders, namely, Duchenne muscular dystrophy; Clinical medical reagents for use in gene therapy; Therapeutic pharmaceutical compositions that include recombinant adeno-associated viral (rAAV) vectors to provide a platform for specific expression of biologics with therapeutic effect in gene therapy; Pharmaceutical preparations containing adeno-associated viral (AAV) vectors and nucleic acids for a system delivering genes to biological cells for use in the treatment of genetic diseases and disorders, namely, Duchenne muscular dystrophy; Gene therapy preparation for medical use containing genetic materials for sustained protein expression, namely, microdystrophin expression
Pharmaceuticals