Translarna™ (ataluren) Slows Disease Progression in Patients with DMD

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 Translarna (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.

«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.»

Researchers 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

Sarepta Therapeutics Receives Complete Response Letter from the US FDA for Golodirsen New Drug Application to treat patients with Duchenne Muscular Dystrophy (DMD)

CAMBRIDGE, Mass., Aug. 19, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced it had received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.

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