Preparations for use in the treatment of autosomal dominant disorders; pharmaceutical preparations, namely, preparations for use in the treatment of rare diseases; pharmaceutical preparations, namely, preparations for use in the treatment of congenital diseases; pharmaceutical preparations, namely, preparations for use in the treatment of Amyotrophic Lateral Sclerosis (ALS); pharmaceutical preparations, namely, preparations for use in the treatment of Alpha-1 Antitrypsin Deficiency; biological preparations for use in the treatment of autosomal dominant disorders, rare diseases, congenital diseases, Amyotrophic Lateral Sclerosis (ALS), and Alpha-1 Antitrypsin Deficiency; gene therapy products for use in the treatment of autosomal dominant disorders, rare diseases, congenital diseases, Amyotrophic Lateral Sclerosis (ALS), and Alpha-1 Antitrypsin Deficiency
Pharmaceuticals