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Therapeutic Advances in Rare Disease|December 2, 2024
Gastrointestinal langerhans cell histiocytosis in an adult presenting with anal protrusion and multiple colorectal ulcers: a case reportNhu Tung Tran, Quoc Thanh Truong, Khuyen Thi Nguyen, et al.Therapeutic Advances in Rare Disease|November 4, 2024
Congenital insensitivity to pain with anhidrosis: a literature review and the advocacy for stem cell therapeutic interventionsMuhammad Ikrama, Muhammad Usama, Muhammad Hassan Haider, et al.Therapeutic Advances in Rare Disease|February 13, 2025
Nirogacestat-the pathway to approval of the first treatment for desmoid tumors, a rare diseaseShivaani Kummar, Nam Bui, Wells A Messersmith, et al.Therapeutic Advances in Rare Disease|September 9, 2024
The small steps that lead to big impact: translating therapeutics from idea to reality for the CDKL5 deficiency disorder communityAmanda Jaksha, Marissa Bishop, Karen Utley, et al.Therapeutic Advances in Rare Disease|July 29, 2024
Patient organization perspective: a research roadmap for Okur-Chung Neurodevelopmental SyndromeGabrielle V Rushing, Jennifer SillsTherapeutic Advances in Rare Disease|July 29, 2024
The role of the TSC Alliance in advancing therapy development: a patient organization perspectiveSteven L Roberds, Zoë Fuchs, Elizabeth M Cassidy, et al.Therapeutic Advances in Rare Disease|May 14, 2023
A Systematic Review investigating the Effectiveness of Exercise training in Glycogen Storage DiseasesClaire Bordoli, Elaine Murphy, Ian Varley, et al.Therapeutic Advances in Rare Disease|May 14, 2023
A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going?Morag Griffin, Richard Kelly, Alexandra PikeTherapeutic Advances in Rare Disease|May 14, 2023
Papilledema and retinopathy lead to diagnosis of IgA nephropathy: a case reportJames T Kwan, Erin Lanzo, David J Ramsey, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Keratoconus: cross-linking the window of the eyeSally Hayes, Siân R Morgan, Keith M MeekPageof 13