GLUCOSYLCERAMIDE SYNTHASE INHIBITORS

The application relates to compounds of formula 1 which act as inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of can...

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Main Authors SKERLJ, RENATO, MARSHALL, JOHN, CHENG, SENG H, LEONARD, JOHN, NATOLI, THOMAS, BOURQUE, ELYSE, XIANG, YIBIN, HIRTH, BRADFORD, JANCSICS, KATHERINE, HUSSON, HERVE, BESKROVNAYA, OXANA, SCHEULE, RONALD K, METZ, MARKUS, CABRERA-SALAZAR, MARIO A, CELATKA, CASSANDRA, ZHAO, ZHONG, MAKINO, ELINA, GOOD, ANDREW
Format Patent
LanguageEnglish
French
Published 17.10.2023
Subjects
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
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Summary:The application relates to compounds of formula 1 which act as inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer. L'invention concerne des inhibiteurs de synthase de glucosylcéramide (GCS) utilisés dans le traitement de maladies métaboliques, comme les maladies de stockage lysosomal, soit seuls soit en combinaison avec une thérapie de substitution enzymatique, ainsi que de maladies cystiques et dans le traitement du cancer.
Bibliography:Application Number: CA20132884144