Categoría: Orphanet Journal of Rare Diseases
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Systemic metabolic remodeling in mucopolysaccharidosis revealed by untargeted urinary metabolomics
Background Methods Results Conclusions …
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A study on the quality of life and burden of informal caregivers of children with metachromatic leukodystrophy in Poland
Background Results Conclusion Clinical trial…
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A study on the quality of life and burden of informal caregivers of children with metachromatic leukodystrophy in Poland
Background Results Conclusion Clinical trial…
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Systemic metabolic remodeling in mucopolysaccharidosis revealed by untargeted urinary metabolomics
Background Methods Results Conclusions …
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Repetitive transcranial magnetic stimulation normalizes cerebrocerebellar loop functional connectivity in spinocerebellar ataxia type 3
Background Methods Results Discussion …
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Long-term treatment with sirolimus for pediatric vascular anomalies
Background Methods Results Conclusions …