Publication: Early treatment with ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy
This case study, published in Acta Myologica, details an ambulant 2-year-old patient with nonsense mutation Duchenne muscular dystrophy (nmDMD), who received early treatment with a mutation-specific therapy in an expanded-access program
Read about the background, clinical presentation, and diagnosis of the patient, who began treatment with a mutation-specific therapy at 2 years of age
Review the clinical outcomes of early treatment with a mutation-specific therapy for nmDMD over 16 months, as measured by assessment of the patient’s motor, cognitive, pulmonary, and cardiac function
Bitetti I, Mautone C, Bertella M, et al. Acta Myologica. 2022;40(4):184–186
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▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC at email@example.com.
Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.
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MED-ALL-DMD-2200118 | May 2022
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