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Causton, H
L.OsterhausG
As seen through initiatives and shared testimonial experiences, the collective advocacy of families can inspire confidence in new approaches and therapies, encouraging others to seek potential solutions for their loved ones
D.RuftyT
Human clinical trial data is extremely limited
Levtchenko E, Servais A, Hulton SA, Ariceta G, Emma F, Game DS, Lange K, Lapatto R, Liang H, Sberro-Soussan R, Topaloglu R, Das AM, Webb NJA, Wanner C (2022) Expert guidance on the multidisciplinary management of cystinosis in adolescent and adult patients
