ArticleTherapeutic advances in neurological disorders2018
One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?
Article in Therapeutic advances in neurological disorders, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed.
- Safety and effectiveness of ataluren in patients with Duchenne muscular dystrophy: single-center experience from Saudi Arabia.The Journal of international medical research · 2024Article
- The BALB/c.mdx62 mouse exhibits a dystrophic muscle pathology and is a model of Duchenne muscular dystrophy.Disease models & mechanisms · 2024Article
- The Importance of Early Treatment of Inherited Neuromuscular Conditions.Journal of neuromuscular diseases · 2024Review
- Treatment with ataluren in four symptomatic Duchenne carriers. A pilot study.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024Article
- Novel Translational Read-through-Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome.Biomedicines · 2022Article
- Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2021Article
- Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2021Article
- Ataluren-Promising Therapeutic Premature Termination Codon Readthrough Frontrunner.Pharmaceuticals (Basel, Switzerland) · 2021Review
- Nonsense suppression therapies in human genetic diseases.Cellular and molecular life sciences : CMLS · 2021Review
- Read-through approach for stop mutations in Duchenne muscular dystrophy. An update.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2021Review
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8 authors.
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Abstract
backgroundAtaluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials but no report exists about real-life experience. PATIENT/
methodswe describe three Italian children with nmDMD treated with ataluren for 1 year. Measurements were made every 3 months and was evaluated the 6-Minute Walking Distance (6MWD).
resultsCase1 involves a patient with a 6MWD at T0 of 360 m, who started ataluren therapy at age 10 years. Case2 is a child who began treatment with ataluren at age 8 years when he had severe ambulatory compromise (6MWD < 75 m at T0). A third patient (case3) had a 6MWD of 320 m when he started ataluren therapy at age 5 years. The best improvement in 6MWD was observed in case3, a patient in whom treatment with ataluren was started much earlier. In case1, ataluren was started relatively late and 6MWD was maintained at a stable level. Surprisingly, we observed a 50% improvement in 6MWD in case2, a patient who began therapy early, but with a severe loss of lower limb muscle function at the time.
conclusionstreatment responses depend on the patient's age and disease severity when therapy was initiated. On the basis of our experience, the main factor that influences the effectiveness seems to be earlier instigation of therapy and positive results may still be achieved in patients with more severe muscle involvement. Interestingly, these three boys with phenotypically different nmDMD provide useful information regarding future therapeutic recommendations for the ataluren administration in real clinical practice.
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