PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
5 of 70 results sorted by: Most Recent
See all results in PubMed (70)-
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
J Patient Rep Outcomes. 2024. PMID: 39535661 Free PMC article. Clinical Trial.
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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
J Neurol. 2024. PMID: 38418563 Free PMC article. Clinical Trial.
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Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.
Mol Genet Metab. 2024. PMID: 38184428 Free article. Clinical Trial.
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Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
J Neurol. 2024. PMID: 38057636 Free PMC article. Clinical Trial.
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Gaucher Disease Types I and III Responded Well to Substrate Reduction Therapy Using Eliglustat.
Intern Med. 2023. PMID: 36889706 Free PMC article.