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 their fate. It is because these families esp. in the UK and the USA founded organizations, connected with each other, fund-raised, and motivated researchers to investigate therapeutic approaches that we can now celebrate the beginning of the clinical trial! However, we have not yet reached our goal. We do not know whether gene therapy will work. And even if it does, it will unfortunately not be able to help all patients with CNM. For this reason we still need your support to help ourselves. There are other promising therapeutic approaches that need financial support today in the same way gene therapy needed it 10 years ago. For this reason we continue to collaborate with the Myotubular Trust in granting scholarships
More information on gene therapy and also links to the press release you can find here.