Gene Therapy: Dramatically improvement on children with myotubular myopathy

The gene therapy developed for children with myotubular myopathy has had astonishing results in clinical trials. Nine children have received the treatment until now and their neuromuscular function has improved dramatically. Most children can now sit on their own, four are now breathing without ventilators and three are making stepping movements. Myotubular Myopathy (MTM) is … Read more…

Another approval for clinical trials for centronuclear myopathies

Today, the Medicines and Healthcare products Regulatory Agency (MHRA) has approved clinical trials of the drug DYN101. This drug regulates the production of the enzyme dynamin 2 in the body. This could lead to an improvement in the symptoms of people with various centronuclear myopathies (CNM) (those with mutations in the genes MTM1 and DNM2). … Read more…

First promising results from gene therapy

On January 4, 2018 Audentes Therapeutics has published first data on the first three patients in the clinical trial on gene therapy on myotubular myopathy, called ASPIRO. This study has begun in September 2018. The presentation on these preliminary results is on the first three of in total 12 patients. These first three pateints received … Read more…

Research Grant for Jim Dowling

Today (26.10.2017) is this year’s research grant from the Myotubular Trust and ZNM – Zusammen Stark! e.V. officially been awarded: Jim Dowling of the Sick Kids Hospital in Toronto receives € 244,000 to investigate the efficacy of several promising drugs that could greatly reduce the muscle weakness of patients with myotubular myopathy. Jim Dowling and … Read more…

Clinical trial for gene therapy has begun!

This week Audentes Therapeutics has announced that the first patient in the USA has been dosed with AT132. Thus the clinical trial for gene therapy has begun. A very important mile stone has been reached. We are very happy and excited about this. It was worth it that parents world wide did not simply accept … Read more…

Vistit to the IGBMC and Dynacure in Strassbourg

On March 24, 2016, members of  ZNM – Zusammen Stark e. V. and The Myotubular Trust met at IGBMC in Strasbourg to visit the laboratory of Jocelyn Laporte, where Myotubular Trust have been funding projects for a number of years. We had a tour of the IGBMC lab with Jocelyn Laporte, Belinda Cowling and Johann … Read more…