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Substancja (substancje)
- 1-deoxynojirimycin hydrochloride
- Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene
- Alglucosidase alfa
- 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 human acid alpha-glucosidase
- Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues
- Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan
- Triheptanoin
- Valproic acid, sodium
- adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene