{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Zhao L"],"funding":["the National Key Research and Development Program of China"],"pagination":["311"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11344408"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["19(1)"],"pubmed_abstract":["Background
An increasing number of clinical trials for new therapeutic strategies are underway or being considered for dystrophinopathy. Having detailed data on the natural progression of this condition is crucial for assessing the effectiveness of new drugs. However, there's a lack of data regarding the long-term data on the natural course and how it's managed in China. In this study, we offer a comprehensive overview of clinical and molecular findings, as well as treatment outcomes in the Chinese population.Methods
Institutional data on all patients with dystrophinopathy from August 2011 to August 2021 were retrospectively reviewed. The data included geographic distribution, age at diagnosis, molecular findings, and treatment options, such as corticosteroids, cardiac inte"],"journal":["Orphanet journal of rare diseases"],"pubmed_title":["Comprehensive analysis of 2097 patients with dystrophinopathy based on a database from 2011 to 2021."],"pmcid":["PMC11344408"],"funding_grant_id":["2022YFC2703600"],"pubmed_authors":["Li H","Li X","Hu C","Zhao L","Qian C","Zhou S","Zhang L","Zhou Y","Wang Y","Shi Y"],"additional_accession":[]},"is_claimable":false,"name":"Comprehensive analysis of 2097 patients with dystrophinopathy based on a database from 2011 to 2021.","description":"Background
An increasing number of clinical trials for new therapeutic strategies are underway or being considered for dystrophinopathy. Having detailed data on the natural progression of this condition is crucial for assessing the effectiveness of new drugs. However, there's a lack of data regarding the long-term data on the natural course and how it's managed in China. In this study, we offer a comprehensive overview of clinical and molecular findings, as well as treatment outcomes in the Chinese population.Methods
Institutional data on all patients with dystrophinopathy from August 2011 to August 2021 were retrospectively reviewed. The data included geographic distribution, age at diagnosis, molecular findings, and treatment options, such as corticosteroids, cardiac inte","dates":{"release":"2024-01-01T00:00:00Z","publication":"2024 Aug","modification":"2026-07-15T05:08:55.507Z","creation":"2024-11-05T16:11:08.392Z"},"accession":"S-EPMC11344408","cross_references":{"pubmed":["39182149"],"doi":["10.1186/s13023-024-03217-7"]}}