Stof(fen)

• 1-deoxynojirimycin hydrochloride
• Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene
• Clenbuterol
• Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase
• Miglustat
• Recombinant adeno-associated viral vector containing a bioengineered capsid serotype AAV-rh74 and a codon-optimised expression cassette to drive the expression of a secretable form of human acid alpha-glucosidase
• Recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene
• Recombinant humaan alfa-glucosidase geconjugeerd met meerdere kopieën van synthetisch bis-mannose-6-fosfaat-tetra-mannose glycaan
• Recombinant human acid alpha-glucosidase
• Recombinant human acid alpha-glucosidase
• Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues
• Triheptanoin
• Valproic acid, sodium
• adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene

Handelsna(a)m(en)

• LUMIZYME
• MYOZYME