A plea for a gene therapy for ALS

1 point

7 years ago

This is a plea for a gene therapy for the TDP-43 pathology in Amyotrophic lateral sclerosis (ALS. There are still no efficacious drugs for ALS.

The ideas sketched out in this plea are based on methods and technologies that have been published since some years now. One striking application of those ideas is the new drug for SMA: Zolgenma. SMA is a related disease to ALS, and the most common genetic cause of infant death.

https://padiracinnovation.org/Blog/2019/02/a-plea-for-a-gene-therapy-for-als

Please, if you know someone in the pharmaceutical industry who could be interested, could you discuss with her/him about the ideas in this document?

Many thanks

Jean-Pierre

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