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…

Family Meeting 2017

The Annual General Meeting and Family Conference of the association CNM – Together Strong! e.V. will take place from May, 5th to 7th in Tambach, Thuringia-Germany. This time, the conference will be only in German language. Here you can find information about the program and application form. We are looking forward to seeing you again! … 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…