GALNS


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GALNS

A gene on chromosome 16q24.3 that encodes N-acetylgalactosamine-6-sulfatase, a lysosomal exohydrolase required for the degradation of glycosaminoglycans, keratan sulfate and chondroitin 6-sulfate.

Molecular pathology
Defects of GALNS cause Morquio A syndrome, a lysosomal storage disease.
References in periodicals archive ?
including, without limitation, statements about: the expectations of revenue and sales related to Naglazyme, Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as a whole; the timing of BioMarin's clinical trials of GALNS, Firdapse, PEG-PAL, BMN-673, BMN-701 and other product candidates; the continued clinical development and commercialization of Aldurazyme, Naglazyme, Kuvan, Firdapse, and its product candidates; and actions by regulatory authorities.
genetic diseases: GALNS for MPS IV and PEG-PAL for phenylketonuria.
Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA; PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU; BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease; and BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers.