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Therapeutic Advances in Rare Disease|July 12, 2023
Surgical resection therapy of a rare presentation of persistent Mullerian duct syndrome: a case reviewIwens Moreira de Faria, Augusto Machado de Souza, Luiz Rodrigues Pereira Júnior, et al.Therapeutic Advances in Rare Disease|May 14, 2023
IMPACT study: measuring the impact of caregiving on families and healthcare professionals of children and adults living with mucopolysaccharidoses in IrelandSuja Somanadhan, Hannah Bristow, Ellen Crushell, et al.Therapeutic Advances in Rare Disease|March 30, 2026
Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophyArif Mitha, Victoria Hodgkinson, Susi Vander Wyk, et al.Therapeutic Advances in Rare Disease|March 6, 2026
A novel therapy for pyridoxine-dependent epilepsy due to biallelic pathogenic variants in ALDH7A1: secondary mitochondrial energy deficiency and improvements of neurodevelopmental outcomes on triheptanoin treatmentAnastasia Ambrose, Morganne McCabe, Shalini Bahl, et al.Therapeutic Advances in Rare Disease|May 14, 2023
What are the benefits of the anti-FGF23 antibody burosumab on the manifestations of X-linked hypophosphatemia in adults in comparison with conventional therapy? A reviewMarie-Hélène Lafage-ProustTherapeutic Advances in Rare Disease|May 14, 2023
'Granulomatosis with polyangiitis after Pfizer vaccination': a case reportFrancis Essien, Jordan Evans, Andrew Kyle, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Telehealth for patients with rare epilepsiesZachary J Kramer, Carrin Brandt, Kathryn Havens, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Racial differences in the dermatological manifestations of tuberous sclerosis complex and the potential effects on diagnosis and careAshley J Pounders, Gabrielle V Rushing, Sonal Mahida, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Pharmacology update: pamidronate for hypertrophic pulmonary osteoarthropathy in palliative careBethany Faust, Aaron Parkinson, Steven J BaumruckerTherapeutic Advances in Rare Disease|May 14, 2023
Clinical evidence of interventions assessed in Friedreich ataxia: a systematic reviewParidhi Jain, Lohit Badgujar, Jelle Spoorendonk, et al.Pageof 13