Glucosylceramide synthase inhibitors

The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in 5 combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

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Main Authors Good, Andrew, Makino, Elina, Cheng, Seng H, Husson, Herve, Leonard, John, Celatka, Cassandra, Marshall, John, Beskrovnaya, Oxana, Scheule, Ronald K, Xiang, Yibin, Skerlj, Renato, Jancsics, Katherine, Cabrera-Salazar, Mario A, Natoli, Thomas, Bourque, Elyse, Zhao, Zhong, Hirth, Bradford, Metz, Markus
Format Patent
LanguageEnglish
Published 18.02.2021
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Abstract The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in 5 combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
AbstractList The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in 5 combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
Author Hirth, Bradford
Husson, Herve
Bourque, Elyse
Scheule, Ronald K
Cheng, Seng H
Metz, Markus
Leonard, John
Beskrovnaya, Oxana
Makino, Elina
Xiang, Yibin
Natoli, Thomas
Skerlj, Renato
Zhao, Zhong
Jancsics, Katherine
Good, Andrew
Cabrera-Salazar, Mario A
Celatka, Cassandra
Marshall, John
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– fullname: Makino, Elina
– fullname: Cheng, Seng H
– fullname: Husson, Herve
– fullname: Leonard, John
– fullname: Celatka, Cassandra
– fullname: Marshall, John
– fullname: Beskrovnaya, Oxana
– fullname: Scheule, Ronald K
– fullname: Xiang, Yibin
– fullname: Skerlj, Renato
– fullname: Jancsics, Katherine
– fullname: Cabrera-Salazar, Mario A
– fullname: Natoli, Thomas
– fullname: Bourque, Elyse
– fullname: Zhao, Zhong
– fullname: Hirth, Bradford
– fullname: Metz, Markus
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Snippet The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases,...
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SubjectTerms CHEMISTRY
HETEROCYCLIC COMPOUNDS
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
Title Glucosylceramide synthase inhibitors
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