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Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History.
Kinane TB, Mayer OH, Duda PW, Lowes LP, Moody SL, Mendell JR. Kinane TB, et al. J Neuromuscul Dis. 2018;5(1):47-58. doi: 10.3233/JND-170272. J Neuromuscul Dis. 2018. PMID: 29278896 Free PMC article. Clinical Trial.
OBJECTIVE: Describe lung function assessed throughout eteplirsen studies 201/202. METHODS: Studies 201/202 included 12 patients treated with eteplirsen over 5 years. ...RESULTS: Age-adjusted mixed-model repeated-measures analysis showed decreases of 2.3% and 2.6% an …
OBJECTIVE: Describe lung function assessed throughout eteplirsen studies 201/202. METHODS: Studies 201/202 included 12 patients treat …
Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy.
Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM; Eteplirsen Investigators and the CINRG DNHS Investigators. Khan N, et al. J Neuromuscul Dis. 2019;6(2):213-225. doi: 10.3233/JND-180351. J Neuromuscul Dis. 2019. PMID: 30856119 Free PMC article.
Eteplirsen-treated patients (n = 74) were amenable to exon 51 skipping and were receiving glucocorticoids. ...CONCLUSIONS: Significant, clinically meaningful attenuation of FVC%p decline was observed in eteplirsen-treated patients versus CINRG DNHS controls....
Eteplirsen-treated patients (n = 74) were amenable to exon 51 skipping and were receiving glucocorticoids. ...CONCLUSIONS: Significan