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…