Dr. Dave goes on to explain its important to understand genetic therapies are not just for genetic ALS. We can use gene therapy to go after sporadic ALS because we know that there are certain proteins that are involved in 90% of ALS. And if we can go after those proteins using gene therapy approaches, we can now go and treat all ALS, and not just a particular genetic subtype. And that's the future of gene therapy. How can we use gene therapy to go after the sporadic forms of ALS?

More genetic testing and counseling will help identify more people living with the disease who could benefit from gene therapies; people like Larry Falivena.

In August 2017, Larry heard those three terrible words: You have ALS. Like most who experience the lengthy diagnosis process, Larry suspected that was the case, but actually, hearing it from his doctor was devastating. A husband and father of two young boys, he had so many plans for the future, or so he thought.

After his diagnosis, Larry was encouraged to get a genetic test even though he had no known family history of ALS, and he says making that decision changed the course of his diseaseand his life. Sure enough, aSOD1mutation was found, a known genetic cause for the disease.

Larry was able to get into an ongoing clinical trial for a treatment of SOD1-ALS called tofersen, now known as Qalsody.

Read more from the original source:
Let's See More Genetic Testing and Counseling - ALS Association

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