Mechanism of Action:
Ataluren works by promoting “readthrough” of premature stop codons (nonsense mutations) in the dystrophin gene, allowing ribosomes to continue translating the mRNA and produce a full-length dystrophin protein. It does this by interacting with the ribosome and influencing the recruitment of tRNAs, effectively suppressing the premature stop codon.
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Delayed Progression:
Ataluren has been shown to slow down the decline in walking ability and respiratory function in patients with nonsense mutation DMD.
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Improved Muscle Function:
Studies suggest that ataluren can improve muscle function and delay disease progression in individuals with DMD.
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Extended Ambulatory Period:
Ataluren may delay the loss of ambulation in patients with nonsense mutation DMD.
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Long-term Effects:
While ataluren is not a cure for DMD, it offers a potential treatment option to slow down the progression of the disease and improve long-term outcomes.
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