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Therapeutic Advances in Rare Disease|September 19, 2024
A roadmap for SHANK3-related Epilepsy Research: recommendations from the 2023 strategic planning workshopMargaret C Savage, Geraldine Bliss, Joseph D Buxbaum, et al.Therapeutic Advances in Rare Disease|April 30, 2025
Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programsNahya AwadaTherapeutic Advances in Rare Disease|May 29, 2024
Linking Angelman and dup15q data for expanded research (LADDER) database: a model for advancing research, clinical guidance, and therapeutic development for rare conditionsSarah Nelson Potter, Elizabeth Reynolds, Katherine C Okoniewski, et al.Therapeutic Advances in Rare Disease|May 23, 2024
Understanding patient, caregiver, and healthcare provider perspectives of the management of long-chain fatty acid oxidation disordersEileen Sullivan Baker, Jennifer Botham, Tasia Rechisky, et al.Therapeutic Advances in Rare Disease|June 21, 2024
Corrigendum to "Proceedings from the MENA Congress for Rare Diseases, 16-19 May 2024, Abu Dhabi, UAE"Therapeutic Advances in Rare Disease|December 22, 2025
Clinical characteristics, treatments, and complications of Kawasaki disease in Peruvian children: a retrospective observational studyNoé Atamari-Anahui, Giancarlo Alvarado-Gamarra, Nadin Conto-Palomino, et al.Therapeutic Advances in Rare Disease|December 28, 2023
The Rare Diseases Clinical Research Network: a model for clinical trial readinessJoanne M Lumsden, Tiina K UrvTherapeutic Advances in Rare Disease|July 23, 2026
Patients' lived experience of thymidine kinase 2 deficiency: the online survey-based assessment of TK2d patient perspectives studyAmel Karaa, Asha Hareendran, Katie Waller, et al.Therapeutic Advances in Rare Disease|September 10, 2026
Addressing unmet needs in rare diseases: A patient-centred vision through INFORM-RDKenji Demesure, Lien Beckers, Wendy Vansteenkiste, et al.Therapeutic Advances in Rare Disease|January 14, 2025
Roadmap to advance therapeutics for SYNGAP1-related disorder: a patient organization perspective from SynGAP Research FundJ Michael Graglia, Aaron J Harding, Kathryn A HeldePageof 13