Found: 4
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Recommendations for patient screening in ultra-rare inherited metabolic diseases: what have we learned from Niemann-Pick disease type C?
- Published in:
- 2019
- By:
- Publication type:
- journal article
Mortality in patients with Sanfilippo syndrome.
- Published in:
- 2017
- By:
- Publication type:
- journal article
A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases.
- Published in:
- Orphanet Journal of Rare Diseases, 2014, v. 9, n. 1, p. 173, doi. 10.1186/s13023-014-0173-x
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- Publication type:
- Article
A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases.
- Published in:
- 2014
- By:
- Publication type:
- journal article