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Pharmaceutical preparations for the treatment of
neuromuscular diseases and disorders, muscular dystrophies,
central nervous system diseases and disorders, genetic
diseases and disorders; pharmaceutical preparations based on
morpholino oligomer technology platform for the treatment of
neuromuscular diseases and disorders, muscular dystrophies,
central nervous system diseases and disorders, genetic
diseases and disorders, and for the regulation of protein
production; pharmaceutical preparations that modulate RNA
expression for the treatment of neuromuscular diseases and
disorders, muscular dystrophies, central nervous system
diseases and disorders, genetic diseases and disorders, and
for the regulation of protein production; gene therapy
products, namely, gene delivery pharmaceuticals;
pharmaceutical preparations for gene therapy, gene editing,
and genome editing; gene therapy products in the nature of
pharmaceuticals for the treatment of neuromuscular diseases
and disorders, muscular dystrophies, central nervous system
diseases and disorders, genetic diseases and disorders;
biological preparations for the treatment of neuromuscular
diseases and disorders, muscular dystrophies, central
nervous system diseases and disorders, genetic diseases and
disorders; viruses, viral vectors and viral constructs for
the treatment of neuromuscular diseases and disorders,
muscular dystrophies, central nervous system diseases and
disorders, genetic diseases and disorders; pharmaceutical
preparations, biological preparations and gene therapy
products in the nature of pharmaceutical and biological
preparations for the treatment of genetic disorders and
diseases