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…

Forschungsstipendium an Jim Dowling

Heute (26.10.2017) ist das diesjährige Forschungsstipendium des Myotubular Trust und von ZNM – Zusammen Stark! offiziell vergeben worden: Jim Dowling vom Sick Kids Hospital in Toronto erhält 244.000 € zur Erforschung der Wirksamkeit von mehreren vielversprechenden Medikamenten, die die Muskelschwäche von Patienten mit myotubulärer Myopathie sehr stark lindern könnte. Jim Dowling und sein Team haben … 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…