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Furthermore, pragmatic randomized trials directly comparing dose escalation versus maintenance of the 0.5-mg dose with longer-term follow-up are required to establish higher-level evidence on comparative effectiveness and tolerability, including rare AEs
Petyuk VA, Chang R, Ramirez-Restrepo M, Beckmann ND, Henrion MYR, Piehowski PD, Zhu K, Wang S, Clarke J, Huentelman MJ, Xie F, Andreev V, Engel A, Guettoche T, Navarro L, De Jager P, Schneider JA, Morris CM, McKeith IG, Perry RH, Lovestone S, Woltjer RL, Beach TG, Sue LI, Serrano GE, Lieberman AP, Albin RL, Ferrer I, Mash DC, Hulette CM, Ervin JF, Reiman EM, Hardy JA, Bennett DA, Schadt E, Smith RD, Myers AJ
Bertholet-Thomas A, Claramunt-Taberner D, Gaillard S, Deschenes G, Sornay-Rendu E, Szulc P, Cohen-Solal M, Pelletier S, Carlier MC, Cochat P, Bacchetta J (2018) Teenagers and young adults with nephropathic cystinosis display significant bone disease and cortical impairment
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