PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
5 of 145 results sorted by: Most RecentSee all results in PubMed (145)
[Long-term outcome of hematopoietic stem cell transplantation in two children with Mucopolysaccharidosis].
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2023. PMID: 37994129 Chinese.
Safety outcomes and patients' preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond.
Orphanet J Rare Dis. 2023. PMID: 37891668 Free PMC article.
Skeletal phenotype amelioration in mucopolysaccharidosis VI requires intervention at the earliest stages of postnatal development.
JCI Insight. 2023. PMID: 37751300 Free article.
Real-world patient data on immunity and COVID-19 status of patients with MPS, Gaucher, and Pompe diseases from Turkey.
Arch Pediatr. 2022. PMID: 35705384 Free PMC article.
Spinal cord compression in patients with mucopolysaccharidosis.
Eur Spine J. 2022. PMID: 35267074