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

Erscheinungsjahr:

2021

Erschienen:

2021

Enthalten in:

Europäisches Patentamt - (2021) vom: 21. Okt. Zur Gesamtaufnahme - year:2021

Sprache:

Englisch

Beteiligte Personen:

VAN DER WAL ERIK [VerfasserIn]
BERGSMA ATZE JACOBUS [VerfasserIn]
PIJNAPPEL WILHELMUS WENCESLAUS MATTHIAS [VerfasserIn]
VAN DER PLOEG ANTJE TJITSKE [VerfasserIn]
REUSER ARNOLDUS [VerfasserIn]

Links:

Volltext [kostenfrei]

Themen:

Sonstige Themen:
615
A61K: Preparations for medical, dental, or toilet purpose (...)
A61P: Specific therapeutic activity of chemical compounds (...)
bio
C12N: Microorganisms or enzymes; compositions thereof; pr (...)
che

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

Patentnummer:

US2021322526

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

EPA014148102