Nonsense mutation Duchenne muscular dystrophy (nmDMD) is a rare, X-linked genetic disorder that results in a decline in function, loss of ambulation and early death due to respiratory or cardiac failure. Ataluren is conditionally approved by the European Medicines Agency for the treatment of ambulatory patients aged ≥ 5 years with nmDMD. Initiation of treatment prior to substantial muscle loss may maximize benefit. It is therefore important to understand the safety and pharmacokinetics (PK) of ataluren in patients aged < 5 years, particularly since ataluren is dosed by weight.

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