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 the lowest dose of the drug called “AT132”.

The results are quite promising. Audentes says in its own press release that the results “exceeded our expectations“. The webcast (without the short movies) and the presentation can be downloaded from the website of Audentes Therapeutics:

All three treated patients improved in their muscle function, albeit to a different grade. Sligth side effects that could be in connection with the treatment were observed (an elevated concentration of Billirubin and higher values of liver enzymes)

We are very happy about these interim data. Now we must be patient and hope that the next results are similarly positive.

The reason why we can be so hopeful is in fact that patient organizations in the UK and in the USA started with funding research projects many years ago. Among this the work of Ana Buj-Bello, which is one base of the gene therapy for MTM1.For this reason we hope also that the projects that we are rit´gth now co-funding together with the Myotubular Trust will be get to this phase too. Thank you to our donors who make this possible!





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