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A plea for a gene therapy for ALS
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
- gus_massa 8y agoSince the document links for a similar therapy for a similar illness, and links a few studies in animal of similar approach, why do you think that big pharma is not planning to do this in a few years?
- JPLeRouzic 8y agoThanks for having read the document and for the comment! To answer your question: It would be a fantastic news. Even the simple fact that a major player is interested in ALS would be awesome, the current and past trials are only done by tiny organizations and their drugs are not that innovative. However the frequency of ALS is the tenth of SMA, so it might be much more difficult to fund.