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…