PubMed Clinical Queries
This tool uses predefined filters to help you quickly refine PubMed searches on clinical or disease-specific topics. To use this tool, enter your search terms in the search bar and select filters before searching.
Results for Clinical Studies: Therapy/Broad
5 of 89 results sorted by: Most Recent
See all results in PubMed (89)-
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.
Toscano A, et al. Mol Genet Metab. 2024. PMID: 38184428 Free article. Clinical Trial.
-
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).
Byrne BJ, et al. J Neurol. 2024. PMID: 38057636 Free PMC article. Clinical Trial.
-
Population Pharmacokinetic Modeling and Determination of Individual Exposure to Avalglucosidase Alfa in Adolescent and Adult Patients With Late-Onset Pompe Disease: Analysis of Pooled Data From Phase I to III Clinical Trials.
Tuffal G, et al. Ther Drug Monit. 2023. PMID: 37556417 Free PMC article.
-
Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry.
Lessard LER, et al. Mol Genet Metab. 2023. PMID: 37285781 Free article.
-
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, et al. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.