Visit to Strassbourg

Today, October 21, Emil was invited to the IGBMC (Institut de génétique et de biologie moléculaire et cellulaire. We got to know the research group of Jocelyne Laporte and Johann Böhm and  explained how life is with myotubular myopathy. Holger reports about a normal day in Emil’s life       Most of those present … Read more…

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…

CNM-MTM Conference in Chicago

The fourth MTM-CNM Family Conference will be held in Chicago on July 24-26, 2015. Holger Fischer will participate at the conference representing our Association. He will meet families and scientist and for sure will come back with a lot of new information and ideas. In case you have questions that you want to address at this … Read more…