Females and children with Anderson-Fabry disease: Diagnosis, monitoring, benefits of enzyme replacement therapy (ERT) and considerations on timing of starting ERT

Expert Opinion on Orphan Drugs

Volumn 1, Issue 4, 2013, Pages 315-330

  Parini, Rossella ^a   Feriozzi, Sandro ^b  

^a SAN GERARDO HOSPITAL   (Italy)

^b BELCOLLE HOSPITAL   (Italy)

Author keywords

Anderson Fabry disease; Children; Early ERT; Early treatment; Enzymatic replacement therapy; Females

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE; AMINO TERMINAL PRO BRAIN NATRIURETIC PEPTIDE; GELATINASE B;

BRIEF PAIN INVENTORY; DEPRESSION; DISEASE COURSE; DISEASE SEVERITY; DIZZINESS; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; ENZYME REPLACEMENT; FABRY DISEASE; FATIGUE; GENE MUTATION; GENETIC ANALYSIS; GLOMERULUS FILTRATION RATE; HEADACHE; HEART FAILURE; HEART PALPITATION; HEART RATE VARIABILITY; HEART VENTRICLE HYPERTROPHY; HUMAN; KIDNEY INJURY; LIBIDO DISORDER; MAINZ SEVERITY SCORE INDEX; MISSENSE MUTATION; NONSENSE MUTATION; NUCLEAR MAGNETIC RESONANCE IMAGING; PHENOTYPE; PULMONARY VALVE INSUFFICIENCY; QUALITY OF LIFE; REVIEW; SCORING SYSTEM; SWEAT TEST; SYSTEMATIC REVIEW; TINNITUS; VERTIGO; X CHROMOSOME INACTIVATION;

EID: 84896724822     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2013.776957     Document Type: Review

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