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A Randomized, Double‐Blind, Placebo‐Controlled, Multiple Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of ELX‐02 in Healthy Subjects.

• Authors : Leubitz, Andi; Vanhoutte, Frederic; Hu, Ming‐yi

Subjects: NONSENSE mutation; PHARMACOKINETICS; CYSTIC fibrosis

• Source: Clinical Pharmacology in Drug Development; Aug2021, Vol. 10 Issue 8, p859-869, 11p

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Cysteamine in renal transplantation: A report of two patients with nephropathic cystinosis and the successful re-initiation of cysteamine therapy during the immediate post-transplant period.

• Authors : Berryhill, Allison; Bhamre, Suvarna; Chaudhuri, Abanti

Subjects: CYSTEAMINE; CYSTINOSIS; TRANSPLANTATION of organs, tissues, etc. in children

• Source: Pediatric Transplantation; Feb2016, Vol. 20 Issue 1, p141-145, 5p

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SSIEM 2014 Annual Symposium: Abstracts.

• Source: Journal of Inherited Metabolic Disease; Sep2014 Supplement, Vol. 37, p27-185, 159p

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Cystinosis myopathy: Searching for optimal clinical outcome measures.

• Authors : Kushlaf, Hani

• Source: Muscle & Nerve; Dec2020, Vol. 62 Issue 6, p652-653, 2p

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