ENZYMATIC REPLACEMENT THERAPY AND ANTISENSE THERAPY FOR POMPE DISEASE
The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene..
Medienart: |
Patent |
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Erscheinungsjahr: |
2021 |
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Erschienen: |
2021 |
Enthalten in: |
Europäisches Patentamt - (2021) vom: 21. Okt. Zur Gesamtaufnahme - year:2021 |
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Sprache: |
Englisch |
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Beteiligte Personen: |
VAN DER WAL ERIK [VerfasserIn] |
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Links: |
Volltext [kostenfrei] |
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Anmerkungen: |
Source: www.epo.org (no modifications made), First posted: 2021-10-21, Last update posted on www.tib.eu: 2023-05-28, Last updated: 2023-06-06 |
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Patentnummer: |
US2021322526 |
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Förderinstitution / Projekttitel: |
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PPN (Katalog-ID): |
EPA014148102 |
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