PTC Therapeutics Announces New Real-World Analysis Demonstrating Translarna(TM) (ataluren) Slows Disease Progression in Patients with Duchenne Muscular Dystrophy

About this update from Ptc Therapeutics, Inc.

[{"type":"text","content":"SOUTH PLAINFIELD, N.J., Oct. 4, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that data from STRIDE,* the first international registry for patients with Duchenne muscular dystrophy due to a nonsense mutation receiving Translarnaä (ataluren), demonstrate that Translarna preserves lung function in children and adolescents compared with a matched cohort in a long-term natural history study.1 The real world analysis was presented at the 24th International Annual Congress of the World Muscle Society.\n\"Across ambulation, physical function and lung function, the STRIDE data demonstrate that patients receiving Translarna preserved function for years longer than patients receiving standard of care,\" said Stuart Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"These are real world results that provide clinicians and regulators the true picture of patient response to treatment.\"\nResearchers evaluated FVC, a traditional measure of lung function in Duchenne patients that correlates with disease progression and mortality.2 The STRIDE data showed that 32.1% of standard of care patients from the natural history cohort had a FVC of <50%, compared to only 2.2% of patients receiving Translarna after a mean total exposure of 633 days.1 The data also indicates that Translarna significantly preserved patients' ability to stand up from lying and climbing stairs compared with natural history.3\nAfter loss of ambulation and loss of the use of the arms, the respiratory muscles of people with Duchenne start to progressively deteriorate, leading to the risk of life-threatening respiratory complications and the need for ventilation support. Patients with a predicted FVC of <50% are considered to be in the late non-ambulatory stage of Duchenne.4 To conduct the analysis, patients from the STRIDE Registry were matched against a comparable cohort of patients from the Cooperative International Neuromuscular Research Group Natural History Study, based on their propensity for disease progression.1\nThese latest data build on the STRIDE registry – time-to-event analysis, which demonstrated that the median age at which patients on Translarna lost the ability to stand up from lying in under 5 seconds (the first key clinically significant Duchenne milestone) is 12 years – 3 years later than seen with natural disease pr...

More updates from Ptc Therapeutics, Inc.