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Audentes commences dosing in Phase I/II trial for myotubular myopathy
US-based biotechnology firm Audentes Therapeutics has commenced patient dosing in the Phase I/II ASPIRO clinical trial of its product candidate AT132 to treat X-linked myotubular myopathy (XLMTM). (Source: Drug Development Technology)
Source: Drug Development Technology - September 21, 2017 Category: Pharmaceuticals Source Type: news

New step toward the treatment of myotubular myopathy gene therapy restores strength and prolongs lives in affected dogs
A team of researchers has demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - April 5, 2017 Category: Science Source Type: news

New step towards the treatment of myotubular myopathy gene therapy restores strength and prolongs lives in affected dogs
(AFM-T é l é thon) A team of researchers in France demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 5, 2017 Category: Global & Universal Source Type: news

Audentes enrols first patient in INCEPTUS study for X-Linked Myotubular Myopathy
Audentes Therapeutics has reported that the first patient has been enrolled in INCEPTUS, a study to characterise the disease presentation in children suffering with X-Linked Myotubular Myopathy (XLMTM). (Source: Drug Development Technology)
Source: Drug Development Technology - August 18, 2016 Category: Pharmaceuticals Source Type: news

JCI early table of contents for Feb. 24, 2014
(Journal of Clinical Investigation) This release contains summaries, links to PDFs, and contact information for the following newsworthy papers to be published online, February 24, 2014: PPAR-γ agonist reverses cigarette smoke induced emphysema in mice; Small molecule-dependent redcution of glutamate improves murine ALS, Increased autophagy associated with BRAF inhibitor resistance; Dynamin 2 as a target for X-linked centronuclear myopathy; Identification of an immune cell trafficking pathway in the CNS, and more. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - February 24, 2014 Category: Cancer & Oncology Source Type: news

In X-Linked Myotubular Myopathy, Enzyme Replacement Therapy Shows Promising Results
A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience. The findings are published in the January edition of Human and Molecular Genetics,* X-linked myotubular myopathy (XLMTM) is a severe muscle disease caused by an absence of a protein called myotubularin. There is currently no treatment for this disorder, and most patients die in infancy or childhood... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - January 24, 2013 Category: Consumer Health News Tags: Bones / Orthopedics Source Type: news

Enzyme replacement therapy shows promising results in X-linked myotubular myopathy
(Medical College of Wisconsin) A collaborative research team including a Medical College of Wisconsin pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 21, 2013 Category: Global & Universal Source Type: news