The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease - A systematic literature review by a European panel of experts. [electronic resource]
Producer: 20191104Description: 212-223 p. digitalISSN:- 1096-7206
- Child
- Enzyme Replacement Therapy
- Europe
- Fabry Disease -- therapy
- Female
- Humans
- Isoenzymes -- therapeutic use
- Male
- Observational Studies as Topic
- Pain -- drug therapy
- Quality of Life
- Randomized Controlled Trials as Topic
- Recombinant Proteins -- therapeutic use
- Treatment Outcome
- Trihexosylceramides -- metabolism
- alpha-Galactosidase -- therapeutic use
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Publication Type: Journal Article; Systematic Review
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