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Updated: Jan 22, 2026

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Evolution of functional class, biochemical and echocardiographic parameters and clinical outcomes after
Herminio Morillas-Climent1, Julia Seller-Moya1, Álvaro Vicedo-López1
1Cardiology Service, Hospital Marina Salud, Denia, Alicante, Spain.
Abstract:
Aim: To analyze the impact of sacubitril/valsartan on functional class, surrogate parameters and clinical outcomes in clinical practice. Methods: Retrospective study of patients with heart failure and reduced ejection fraction that started treatment with sacubitril/valsartan. Results: 149 patients (70.7 ± 9.6 years) were included. At baseline, 83.9, 15.4 and 0.7% were taking sacubitril/valsartan 24/26, 49/51 and 97/103 mg, respectively. After 316.1 ± 155.9 days, these numbers moved to 38.9, 39.6, 12.8% (8.7% discontinued). Sacubitril/valsartan improved functional class (from 2.3 ± 0.6 to 1.8 ± 0.5; p < 0.001), increased ejection fraction (from 31.2 ± 7.0 to 37.3 ± 10.5%; p < 0.001) and reduced NT-proBNP (from 3884 ± 4871 to 1975.3 ± 3006.6 pg/ml; p = 0.0001). Rates of any event, cardiovascular death and heart failure hospitalization/decompensation were 13.2 events/100 patient-years. Conclusion: Sacubitril/valsartan is effective and safe in routine practice.
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