Pharmaceutical preparations and gene therapy products in the nature of injectable biologic preparations for genetic, neurological, motor neuron, or neuromuscular diseases, disorders and conditions; pharmaceutical preparations and gene therapy products in the nature of biological preparations containing genetic materials for treating spinal muscular atrophy, loss of motor neurons, muscle degeneration, muscle weakness, and paralysis; gene therapy preparation in the nature of biological preparations containing genetic materials for sustained survival motor neuron protein expression
Pharmaceuticals