http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3344337-A1
Outgoing Links
Predicate | Object |
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assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_0c4322cb4de342be2dbedcbe0c278f6f |
classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y302-0102 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K45-06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P3-08 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P3-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-47 |
classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-47 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P3-00 |
filingDate | 2016-08-31-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_992053c06bc0d14dffbc78f6239f3f06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_91f47c38c66802fc1bdc907a1e27bac0 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b1be01076ab0adfd17d15adcce94e029 |
publicationDate | 2018-07-11-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber | EP-3344337-A1 |
titleOfInvention | Methods and compositions for the treatment of cytopplasmic glycogen storage disorders |
abstract | The present disclosure is directed to methods of treating a cytoplasmic glycogen storage disorder, including glycogen storage disease I, glycogen storage disease III, glycogen storage disease IV, and/or conditions associated with a PRKAG2 mutation, by administering a lysosomal enzyme such as acid alpha-glucosidase. Conditions associated with a PRKAG2 mutation may include hypotonia, cardiomyopathy, myopathy, cytoplasmic glycogen accumulation, ventricular hypertrophy, severe infantile hypertrophic cardiomyopathy, heart rhythm disturbances, increased left ventricular wall thickness, ventricular pre-excitation, or a combination thereof. Methods of treating a cytoplasmic glycogen storage disorder by administering a lysosomal enzyme and a second therapeutic agent are also described. Other embodiments are directed to methods of treating a cytoplasmic glycogen storage disorder by administering a therapeutic agent as an adjunctive therapy to lysosomal enzyme replacement therapy. |
priorityDate | 2015-08-31-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 1359.