PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
5 of 1,157 results sorted by: Most RecentSee all results in PubMed (1,157)
[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.
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy.
Mol Ther Methods Clin Dev. 2023. PMID: 37920237 Free PMC article.
A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT.
Orphanet J Rare Dis. 2023. PMID: 37915038 Free PMC article. Clinical Trial.
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.
Tracheal Narrowing and Its Impact on Anesthesia Care in Patients With Morquio A (Mucopolysaccharidosis Type IVA): An Observational Study.
Anesth Analg. 2023. PMID: 37862598