Audentes Therapeutics Announces Publication of Data from RECENSUS, a Retrospective Medical Record Review of Patients With X-Linked Myotubular Myopathy Tue, 21 Nov 2017 13:00:00 +0000 SAN FRANCISCO, Nov. 21, 2017 /PRNewswire/ -- Audentes Therapeutics, Inc. (BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced the publication of "A Multicenter, Retrospective Medical Record Review of X-Linked Myotubular Myopathy: The RECENSUS Study," in Muscle & Nerve. This publication from RECENSUS, a study sponsored by Audentes, expands upon the data first presented at the Muscular Dystrophy Association (MDA) Scientific Conference in March 2017, and reinforces the high mortality and significant disease burden experienced by children with XLMTM and their families. 32% of patients >18 months of age). At birth, 95% of the boys were hypotonic and 90% required some form of respiratory support. After the immediate neonatal period, once patients have been stabilized, almost half (48%) required 24-hour ventilation, while the remaining boys averaged 8.5 hours per day on a ventilator. 60% of the boys in the study had undergone a tracheostomy. Critically, the data show that infants with XLMTM spend, on average, 35% of their first year of life in the hospital and undergo an average of 3.7 surgeries during that time.
Audentes Therapeutics to Present at Upcoming Investor Conferences Thu, 16 Nov 2017 13:00:00 +0000 SAN FRANCISCO , Nov. 16, 2017 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with ...
Audentes Therapeutics reports 3Q loss Thu, 16 Nov 2017 10:14:31 +0000 On a per-share basis, the San Francisco-based company said it had a loss of 88 cents. The results fell short of Wall Street expectations. The average estimate of five analysts surveyed by Zacks Investment ...
Audentes Therapeutics, Inc. to Host Earnings Call Tue, 14 Nov 2017 17:00:00 +0000 NEW YORK, NY / ACCESSWIRE / November 14, 2017 / Audentes Therapeutics, Inc. (NASDAQ: BOLD ) will be discussing their earnings results in their Q3 Earnings Call to be held on Tuesday, November 14, 2017 ...
Audentes Therapeutics Announces Rare Pediatric Disease and Fast Track Designations for AT132 for the Treatment of X-Linked Myotubular Myopathy Wed, 27 Sep 2017 12:00:00 +0000 SAN FRANCISCO, Sept. 27, 2017 /PRNewswire/ -- Audentes Therapeutics, Inc. (BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease and Fast Track designations for AT132, the company's gene therapy product candidate being developed to treat X-Linked Myotubular Myopathy (XLMTM). In addition to these two new designations, AT132 has also received Orphan Drug designation from both the FDA and European Medicines Agency.
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