Galactosialidosis: historic aspects and overview of investigated and emerging treatment options

Expert Opinion on Orphan Drugs

Volumn 5, Issue 2, 2017, Pages 131-141

  Annunziata, Ida ^a   d’Azzo, Alessandra ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

CTSA; galactosialidosis; lysosomal storage disease; PPCA; therapy

Indexed keywords

BETA GALACTOSIDASE; CARBOXYPEPTIDASE C; NEURAMINIDASE 1; PROTECTIVE PROTEIN CATHEPSIN A; SERINE CARBOXYPEPTIDASE; SIALIDASE; UNCLASSIFIED DRUG;

ADULT DISEASE; CTSA GENE; GALACTOSIALIDOSIS; GENE; GENE MUTATION; HISTORY; HUMAN; INFANT DISEASE; JUVENILE; MOUSE MODEL; NONHUMAN; PHENOTYPE; PLEIOTROPY; PRIORITY JOURNAL; PROTEIN DEFECT; PROTEIN SYNTHESIS; REVIEW;

EID: 85010002165     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1080/21678707.2016.1266933     Document Type: Review

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