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Therapeutic Advances in Rare Disease|May 23, 2024
Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregiversMarielle G Contesse, Rebecca J Woods, Mindy Leffler, et al.Therapeutic Advances in Rare Disease|May 27, 2024
Corrigendum to "The Rare Diseases Clinical Research Network: a model for clinical trial readiness"Therapeutic Advances in Rare Disease|February 3, 2026
Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trialsHenning Andersen, Jordi Díaz-Manera, Ozlem Goker-Alpan, et al.Therapeutic Advances in Rare Disease|December 18, 2024
Hutchinson-Gilford progeria syndrome: unraveling the genetic basis, symptoms, and advancements in therapeutic approachesAkhil Arun, Athira Rejith Nath, Bonny Thankachan, et al.Therapeutic Advances in Rare Disease|September 17, 2024
WITHDRAWAL: Proceedings from the MENA Congress for Rare Diseases, 16-19 May 2024, Abu Dhabi, UAETherapeutic Advances in Rare Disease|November 13, 2023
Study protocol of the HD-MED study aiming to personalize drug treatment in Huntington's disease: a longitudinal, observational study to assess medication use and efficacy in relation to pharmacogeneticsStephanie Feleus, Maaike van der Lee, Jesse J Swen, et al.Therapeutic Advances in Rare Disease|August 14, 2023
A multidisciplinary approach to treating a unique case of recurrent metastatic thymic carcinoma: case reportCarol Wang, David Erick Elkowitz, Michael John Esposito, et al.Therapeutic Advances in Rare Disease|April 1, 2026
Current therapeutic landscape of β-thalassemia: focus on gene therapyAaron N Cheng, Janet L KwiatkowskiTherapeutic Advances in Rare Disease|March 26, 2026
Challenges and opportunities for the use of telehealth in rare disease diagnosis, treatment, research, and education: key opinion leader interviews by the IRDiRC telehealth task forceMelissa A Parisi, Adam L Hartman, Mary Catherine V Letinturier, et al.Therapeutic Advances in Rare Disease|May 29, 2025
From roadmap to a sustainable end-to-end individualized therapy pathwayAnneliene H Jonker, Elena-Alexandra Tataru, David P Dimmock, et al.Pageof 13