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…

Overview on our association

We have created a new poster with the important facts of our association. Take a look! Since our foundation in June 2015 until February 2019, the number of members has risen sharply from 28 to 133 people. We represent people from Germany, the Netherlands and Austria. More than two-thirds of our members have a MTM1 … Read more…

ZNM – Zusammen Stark! e. V. announces co-operation with RYR1-foundation

ZNM – Zusammen Stark! e. V. and  the RYR-1 Foundation (Pittsburgh, PA, USA) are excited to announce a collaboration to further strengthen their shared interest in helping those with certain forms of congenital muscle disease. Thanks to our donors, ZNM- Zusammen Stark! e. V.  could recently donate 8,000 Euros to the RYR-1 Foundation, with those funds … Read more…

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…

Newsletter published

Ab jetzt kann der aktuelle Newsletter, die Ausgabe 3 von 2017, von unserer Seite heruntergeladen werden! Wir berichten über die vielen Aktivitäten im Verein, von unserer Familienkonferenz, von den neuesten Ergebnissen aus der Forschung und wie wir dank unserer Spenderinnen und Spender zum ersten Mal einen wesentlichen Beitrag zur Forschung leisten konnten! Und natürlich stellen … 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…