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Research News


Audentes Therapeutics Reports Second Quarter 2018 Financial Results and Provides Update on ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy

Audentes Therapeutics Reports Second Quarter 2018 Financial Results and Provides Update on ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy

Audentes Therapeutics announced continuing positive interim data from the group of patients receiving the first dose of the gene therapy clinical trial ASPIRO, including muscle biopsy results from the first three patients treated in the study and the week 24 efficacy...

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Audentes Therapeutics announces continuing positive data from first dose cohort of ASPIRO, a phase 1/2 clinical trial of AT132 in patients with x-linked myotubular myopathy (MTM1)

Audentes Therapeutics announces continuing positive data from first dose cohort of ASPIRO, a phase 1/2 clinical trial of AT132 in patients with x-linked myotubular myopathy (MTM1)

Audentes Therapeutics announced continuing positive interim data from the group of patients receiving the first dose of the gene therapy clinical trial ASPIRO. Three more patients have been added to this first dose cohort and Audentes Therapeutics reported: -...

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Grant Award Announced

Grant Award Announced

Dr James Dowling is announced as recipient of 2017 Myotubular Trust award Combinatorial therapy as a novel treatment strategy for myotubular myopathy We are delighted to announce a three year grant award to Dr James Dowling of Sick Kids Toronto. Two...

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Gene Therapy Clinical Trials Begin

Gene Therapy Clinical Trials Begin

Audentes Therapeutics, the San Francisco based biotechnology company who are taking virus mediated gene therapy to clinical trial, have today announced that the first patient has been dosed. Preliminary results will be announced in early January. Read the press...

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Collaboration With Leading Research Charity

We are delighted to announce an exciting and new collaboration with the renowned children’s medical research charity Action Medical Research. They are joining us in the quest to find treatments for myotubular and centronuclear myopathy. Since founding in 1952 Action...

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Study of affected female carriers

Study of affected female carriers

French study published on largest ever cohort of x-linked female carriers. Read the full publication here. It was thought that female carriers are usually asymptomatic but seldom show muscle weakness. The molecular, pathological and phenotypic spectrum of female...

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First Ever Clinical Trial Approved

First Ever Clinical Trial Approved

FDA give go ahead for clinical trials in gene therapy for x-linked myotubular myopathy Audentes Therapeutics today announce approval by the US FDA (Food and Drug Administration) for their application for gene therapy human clinical trials. The approval means they can...

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RECENSUS study data announced

RECENSUS study data announced

At the 2017 Muscular Dystrophy Scientific Conference in Virginia, US  "A Multicenter, Retrospective Medical Record Review of Patients with X-Linked Myotubular Myopathy (XLMTM): The RECENSUS Study" (RECENSUS poster_MDA 2017_v4.2_final copy) was presented by Alan Beggs,...

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New Gene Identified

With the support of funding from the Myotubular Trust, the team of Jocelyn Laporte, have identified a novel gene - CACNA1S - implicated in centronuclear myopathy. This is a very important breakthrough as it highlights new potential research avenues, and is in fact a...

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Research grant announced 2016

Research grant announced 2016

Myotubular Trust is delighted to announce a third year’s grant to Dr James Dowling at Sick Kids’ Hospital, Toronto for a very promising research project, developing at least one treatment option for myotubular myopathy. Dr Dowling was a recipient of 2014 & 2015...

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Dr James Dowling Publication

Dr James Dowling Publication

Dr James Dowling of Sick Kids, Toronto, Canada has been a recipient of grants from the Myotubular Trust in both 2014 and 2015 to research a potential treatment for myotubular myopathy. Dr Dowling has been working on proving the effect of reducing levels of the...

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Outstanding Award Dr Ana Buj Bello, Myotubular Trust grant recipient, receives Outstanding New Investigator Award from the American Society of Gene & Cell Therapy

Outstanding Award Dr Ana Buj Bello, Myotubular Trust grant recipient, receives Outstanding New Investigator Award from the American Society of Gene & Cell Therapy

Dr Ana Buj Bello, head of the Neuromuscular Disorders team at Genethon, Paris, and recipient of two major research grants from the Myotubular Trust in 2008* & 2012**, received the prestigious award of Outstanding New Investigator from the American Society of Gene...

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New, Groundbreaking Research Grant Funded by Myotubular Trust Offers A Huge Leap Forward In Understanding MTM And CNM Helping Find Potential New Therapeutic Treatments

New, Groundbreaking Research Grant Funded by Myotubular Trust Offers A Huge Leap Forward In Understanding MTM And CNM Helping Find Potential New Therapeutic Treatments

The Myotubular Trust is delighted to announce a fourth successive year of making substantial research grants to help find a treatment or cure for myotubular and centronuclear myopathy. Following a rigorous application and peer review process, our Scientific Advisory...

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Two New Research Projects Funded

Two New Research Projects Funded

Myotubular myopathies (MTM) are rare diseases which cause severe muscle weakness and particularly target the muscles which are essential to maintain life i.e. to breathe and eat. Other organs such as the liver are also affected. The most severe form of the disease is...

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