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PTC Therapeutics Announces First Publication of Real-World Data Showing Translarnaä (ataluren) Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy
- STRIDE registry analysis shows Translarna preserved ambulation and physical function by years compared with those in CINRG Duchenne Natural History Study,
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- STRIDE registry analysis shows Translarna preserved ambulation and physical function by years compared with those in CINRG Duchenne Natural History Study, with no new safety signals1-\n - Trend toward delayed worsening of pulmonary function compared with natural history study1-\n\n\nSOUTH PLAINFIELD, N.J., Feb. 3, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Pulmonary function was also preserved in those treated with Translarna.1 The analysis, presented in the publication of an interim analysis of preliminary real-world data, compared children treated with Translarna in a real-world setting from the STRIDE[*] registry with a matched cohort in a long-term natural history study, CINRG[†].1 In addition, no new safety signals were observed in the patients treated with Translarna, consistent with what has been shown in previous clinical trials.1 The interim data have been published in the Journal for Comparative Effectiveness Research. Final data from the STRIDE registry is expected in 2025.\n\n \n \n\n \n\"Duchenne muscular dystrophy is a devastating disease that causes irreversible muscle wasting and progressively robs young people of their ability to walk, move, and breathe naturally without a ventilator, and it reduces their autonomy in daily life tasks,\" said Dr. Andrés Nascimento, Pediatric Neurology, Neuromuscular Diseases Unit, SJD Children's Hospital, Barcelona, Spain. \"In a real-world setting, children and adolescents treated with Translarna experience a delay in the disease progression, are able to maintain more mobility, and have a higher level of physical autonomy concerning the course of the natural history of the disease. This is not only clinically relevant, but especially important for the quality of life of patients and their families.\" \nChildren treated with Translarna in a real-world setting as part of the STRIDE registry were able to walk independently for an additional 3.5 years compared with a propensity-score matched cohort in the CINRG natural history study, with a median age at loss of ambulation of 14.5 years and 11 years, respectively (72% relative risk r...