PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
5 of 1,354 results sorted by: Most RecentSee all results in PubMed (1,354)
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
J Med Genet. 2023. PMID: 37940383 Free article.
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.
Effectiveness and Safety of Eliglustat Treatment in Gaucher Disease: Real-life Unicentric Experience.
Clin Ther. 2023. PMID: 37722956 Free article.
Screening data from 19 patients with late-onset Pompe disease for a phase I clinical trial of AAV8 vector-mediated gene therapy.
JIMD Rep. 2023. PMID: 37701327 Free PMC article.
Patient-reported outcomes in Gaucher's disease: a systematic review.
Orphanet J Rare Dis. 2023. PMID: 37626429 Free PMC article.