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Novel TMEM98, MFRP, PRSS56 variants in a large United States high hyperopia and nanophthalmos cohort von Prasov, Lev, Guan, Bin, Ullah, Ehsan, Archer, Steven M., Ayres, Bernadete M., Besirli, Cagri G., Wiinikka-Buesser, Laurel, Comer, Grant M., Del Monte, Monte A., Elner, Susan G., Garnai, Sarah J., Huryn, Laryssa A., Johnson, Kayla, Kamat, Shivani S., Lieu, Philip, Mian, Shahzad I., Rygiel, Christine A., Serpen, Jasmine Y., Pawar, Hemant S., Brooks, Brian P., Moroi, Sayoko E., Richards, Julia E., Hufnagel, Robert B.

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416P Effect of ataluren on upper limb function in nmDMD patients from the STRIDE Registry von Muntoni, F., Buccella, F., Osorio, A. Nascimento, Tulinius, M., de Resende, M. Bernadete Dutra, Johnson, S., Werner, C., Anbu, B., Liu, E., Mercuri, E.

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395P Age at loss of ambulation in patients with nmDMD from the STRIDE Registry: sensitivity analyses von Muntoni, F., Buccella, F., Osorio, A. Nascimento, Tulinius, M., de Resende, M. Bernadete Dutra, Johnson, S., Werner, C., Anbu, B., Liu, E., Mercuri, E.

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Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim... von Mercuri, Eugenio, Osorio, Andrés Nascimento, Muntoni, Francesco, Buccella, Filippo, Desguerre, Isabelle, Kirschner, Janbernd, Tulinius, Már, de Resende, Maria Bernadete Dutra, Morgenroth, Lauren P., Gordish-Dressman, Heather, Johnson, Shelley, Kristensen, Allan, Werner, Christian, Trifillis, Panayiota, Henricson, Erik K., McDonald, Craig M.

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Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry (S34.008) von McDonald, Craig M., Mercuri, Eugenio, Muntoni, Francesco, Gordish-Dressman, Heather, Morgenroth, Lauren P., de Resende, Maria Bernadete Dutra, Zhou, Shuizhen, Neeharika, Mathukumalli Lakshmi, Haginoya, Kazuhiro, Ramos-Platt, Leigh, Williams, Paula, Penematsa, Vinay, Chou, Connie, Lin, Min, Johnson, Shelley, Koladicz, Karyn, Mastrandrea, Nicholas, Werner, Christian, Trifillis, Panayiota

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