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STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy
- Translarna was seen to slow disease progression compared to standard of care alone - - Data from long-term, real-world results from 241 patients in the
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Translarna was seen to slow disease progression compared to standard of care alone -\n - Data from long-term, real-world results from 241 patients in the STRIDE* patient registry -\n\n\nSOUTH PLAINFIELD, N.J., Sept. 20, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care (SoC) alone. Pulmonary function decline was also delayed by 1.8 years in those treated with Translarna and SoC.\n\n \n \n \n \n \n \n\n \n\"The five-year analysis of the STRIDE registry clearly demonstrates Translarna's profound impact on changing the course of disease progression, said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. \"We are all proud to see Translana's life-changing effect on boys with Duchenne. The results robustness showing both a five-year delay in loss of walking as well as its ability to prolong lung function confirms what we have seen in our clinical trials. These results add to the totality of the evidence of Translarna's benefit for the patients and their families.\"\nA time-to-event analysis of five years of registry data, presented at the World Muscle Society (WMS) 2021 Virtual Congress, shows that boys treated with Translarna plus SoC had a median age of loss of ambulation of 17.9 years old compared with 12.5 years old for those on SoC alone.1 At 12 years old, 80% of boys receiving Translarna plus SoC are still walking, compared to 52% of the boys receiving SoC alone.1 \nThe median age that boys treated with Translarna reached a predicted forced vital capacity (FVC) lower than 60% was 17.6 years old, compared with 15.8 years old for those who did not receive Translarna. This delay in loss of lung function is critical, as the sub-60% threshold is considered the milestone at which patients usually start to require respiratory physical therapy.\n\"Watching your child losing his abilities, until he can no longer walk or even breathe without help is heartbreaking,\" said Filippo Buccella, patient advocate and founder of Parent Project Italy. \"We are starting to see more evidence that Translarna can potentially give children many more years o...