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The IAAM LTBP4 Haplotype is Protective Against Dystrophin-Deficient Cardiomyopathy.
Bello L, Sabbatini D, Fusto A, Gorgoglione D, Borin GU, Penzo M, Riguzzi P, Villa M, Vianello S, Calore C, Melacini P, Vio R, Barp A, D'Angelo G, Gandossini S, Politano L, Berardinelli A, Messina S, Vita GL, Pedemonte M, Bruno C, Albamonte E, Sansone V, Baranello G, Masson R, Astrea G, D'Amico A, Bertini E, Pane M, Lucibello S, Mercuri E, Spurney C, Clemens P, Morgenroth L, Gordish-Dressman H, McDonald CM, Hoffman EP; CINRG-DNHS Investigators; Pegoraro E. Bello L, et al. Among authors: hoffman ep. J Neuromuscul Dis. 2024;11(2):285-297. doi: 10.3233/JND-230129. J Neuromuscul Dis. 2024. PMID: 38363615 Free PMC article.
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T; CINRG DNHS Investigators; Hoffman EP. Clemens PR, et al. Among authors: hoffman ep. J Neuromuscul Dis. 2023;10(3):439-447. doi: 10.3233/JND-221656. J Neuromuscul Dis. 2023. PMID: 37005891 Free PMC article. Clinical Trial.
Reply to F. Muntoni et al.: "In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Clemens PR, Hoffman EP. Clemens PR, et al. Among authors: hoffman ep. J Neuromuscul Dis. 2023;10(6):1155-1157. doi: 10.3233/JND-239005. J Neuromuscul Dis. 2023. PMID: 37955094 Free PMC article. Clinical Trial. No abstract available.
Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022.
Phung K, Crabtree N, Connolly AM, Furlong P, Hoffman EP, Jackowski SA, Jayash SN, Johnson A, Koujok K, Munns CF, Niks E, Rauch F, Schrader R, Turner C, Vroom E, Weber DR, Wong BL, Guglieri M, Ward LM, Wong SC. Phung K, et al. Among authors: hoffman ep. J Neuromuscul Dis. 2024;11(1):233-252. doi: 10.3233/JND-230176. J Neuromuscul Dis. 2024. PMID: 37980681 Free PMC article. No abstract available.
226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20-22 January 2017, Heemskerk, The Netherlands.
Aartsma-Rus A, Ferlini A, McNally EM, Spitali P, Sweeney HL; workshop participants. Aartsma-Rus A, et al. Neuromuscul Disord. 2018 Jan;28(1):77-86. doi: 10.1016/j.nmd.2017.10.002. Epub 2017 Oct 26. Neuromuscul Disord. 2018. PMID: 29203356 Free PMC article. No abstract available.
565 results