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…

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…

European Conference

On May 07. and 08., 2016 the European family conference on myotubular myopathy and other centronuclear myopathies takes place in Niedernhausen near Frankfurt, Germany. It is organized together by the Myotubular Trust and ZNM – Zusammen Stark! Information on the conference, the detailed program (incl workshops) and to the registration process you will find here.  … Read more…

Family Conference – Save-the-Date – May 7

  ZNM – Zusammen Stark! e. V. and the Myotubular Trust wil host the next family conference on myotubular myopathy and other centronuclear myopathies on May 07 in Germany near Frankfurt. More information you will find soon on our website: Family conference 2016. We hope that many of you can come!

EMA grants orphan designation to MTM-Treatment

The European Medicine Agency recently granted orphan designation* to the virus containing MTM1 for the gene therapy for x-linked  myotubular myopathy (XLMTM).  To read more about this designation, please go to the Website of the EMA. *A status assigned to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine … Read more…