Courier allows specific children with DMD to walk for 5 more years!

▎The editor of WuXi AppTec's content team watched your child lose normal mobility until he can no longer walk or even breathe without help.
This is very heartbreaking
.

Recently, PTC Therapeutics announced that the small molecule therapy Translarna (ataluren) has obtained positive results in a long-term real-world study
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Data from 241 patients indicate that compared with standard treatment (SoC) alone, Translarna can delay the inability to get out of bed in boys with nonsense mutant Duchenne muscular dystrophy (nmDMD) by more than 5 years, and the decline in lung function is also delayed on average 1.
8 years
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Duchenne muscular dystrophy mainly affects men.
It is a rare and fatal genetic disease that causes progressive muscle weakness from early childhood.
Patients often die prematurely due to heart and respiratory failure in their 20s
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This progressive muscle disease is caused by a lack of functional dystrophin
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In about 13% of patients with Duchenne muscular dystrophy, nonsense mutations cause the appearance of a stop codon, which leads to the premature termination of the protein synthesis process and the production of dystrophin with loss of function
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Translarna combines with the stop codon to allow the ribosome responsible for translating RNA to add an amino acid where it reads the stop codon, instead of stopping the translation process, thereby restoring normal protein production
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▲Translarna's mechanism of action (picture source: PTC Therapeutics official website) The analysis of this 5-year real-world data released at the World Muscle Association (WMS) conference in 2021 shows that patients receiving Translarna and standard treatment lose their ability to get out of bed.
The median age was 17.
9 years, and the control group was 12.
5 years old
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At the age of 12, 80% of patients receiving Translarna plus standard treatment were still walking, compared with 52% in the control group
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In addition, the median age at which patients receiving Translarna treatment reached a predicted forced vital capacity (FVC) of less than 60% was 17.
6 years, and the control group was 15.
8 years
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The delay in the loss of lung function is critical, because a threshold below 60% is considered a sign that the patient usually starts to require respiratory physical therapy
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Dr.
Stuart W.
Peltz, Chief Executive Officer of PTC Therapeutics, said: "The five-year analysis of this study clearly demonstrates the profound impact of Translarna on changing the course of the disease
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We are proud to see Translana change the lives of children with Duchenne muscular dystrophy.
Effect
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The results of this long-term real-world study further confirm the results we have observed in clinical trials and add to the evidence that Translarna benefits patients and their families
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"Reference: [1] STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy.
Retrieved September 20, 2021, from https:// /stride-data-show-translarna-delays-loss-of-ambulation-by-more-than-five-years-in-boys-with-nonsense-mutation-duchenne-muscular-dystrophy-301380360.
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