Found: 9
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Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.
- Published in:
- 2014
- By:
- Publication type:
- journal article
Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications.
- Published in:
- 2014
- By:
- Publication type:
- journal article
Clinical pathways for inborn errors of metabolism: warranted and feasible.
- Published in:
- Orphanet Journal of Rare Diseases, 2013, v. 8, n. 1, p. 1, doi. 10.1186/1750-1172-8-37
- By:
- Publication type:
- Article
Cost-effectiveness of enzyme replacement therapy for Fabry disease.
- Published in:
- Orphanet Journal of Rare Diseases, 2013, v. 8, n. 1, p. 1, doi. 10.1186/1750-1172-8-29
- By:
- Publication type:
- Article
Cost-effectiveness of enzyme replacement therapy for Fabry disease.
- Published in:
- 2013
- By:
- Publication type:
- journal article
Clinical pathways for inborn errors of metabolism: warranted and feasible.
- Published in:
- 2013
- By:
- Publication type:
- Letter
Tetrahydrobiopterin responsiveness in phenylketonuria: prediction with the 48-hour loading test and genotype.
- Published in:
- 2013
- By:
- Publication type:
- journal article
Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.
- Published in:
- 2011
- By:
- Publication type:
- journal article
"MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial.
- Published in:
- 2011
- By:
- Publication type:
- journal article