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Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.
Ayyar Gupta V, Pitchforth JM, Domingos J, Ridout D, Iodice M, Rye C, Chesshyre M, Wolfe A, Selby V, Mayhew A, Mazzone ES, Ricotti V, Hogrel JY, Niks EH, de Groot I, Servais L, Straub V, Mercuri E, Manzur AY, Muntoni F; iMDEX Consortium and the U.K. NorthStar Clinical Network. Ayyar Gupta V, et al. Among authors: pitchforth jm. PLoS One. 2023 Apr 26;18(4):e0283669. doi: 10.1371/journal.pone.0283669. eCollection 2023. PLoS One. 2023. PMID: 37099511 Free PMC article.
Rimeporide as a first- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy.
Previtali SC, Gidaro T, Díaz-Manera J, Zambon A, Carnesecchi S, Roux-Lombard P, Spitali P, Signorelli M, Szigyarto CA, Johansson C, Gray J, Labolle D, Porte Thomé F, Pitchforth J, Domingos J, Muntoni F. Previtali SC, et al. Among authors: pitchforth j. Pharmacol Res. 2020 Sep;159:104999. doi: 10.1016/j.phrs.2020.104999. Epub 2020 Jun 12. Pharmacol Res. 2020. PMID: 32535224 Free PMC article. Clinical Trial.