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FDA approves treatment of ALS associated with a mutation in the SOD1 gene

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On April 25, 2023, the US Food and Drug Administration (FDA) announced that Qalsody (tofersen) HAS BEEN APPROVED as a new prescription therapy for the treatment of people with rare SOD1-ALS.

The Golden West Chapter is grateful to have worked closely with our 2020 Commitment to Cures honoree, Biogen, and many of our families to provide direct feedback and personal stories that ultimately helped contribute to this outcome. The Golden West Chapter mobilized ALS advocates who participated in several meetings with FDA officials, and submitted our own statement in support.

For people living with ALS and their loved ones, we know that you likely have questions about this new medication, including when it will become available for people with SOD1-ALS. As always, we encourage you to speak directly with your physician about whether or not any medication is right for you.

In addition, please visit our website for the most up-to-date information on Qalsody (tofersen), as well as our other helpful resources about genetic testing and genetic counseling.

We thank everyone who worked tirelessly for more than two decades to make a treatment option for adults living with this rare type of ALS a reality. We are especially proud of our 2014 Essey Award honorees – Golden West Chapter Advisory Trustee, Richard Smith, MD; Frank Bennett, PhD; and Don W. Cleveland, PhD – for their commitment to the development of antisense oligonucleotide (ASO) therapies and extraordinary efforts which led us to this historic day.

This is a monumental milestone, and it was powered by people like YOU – people living with ALS and their loved ones, donors and advocates, researchers, clinicians, industry partners, federal agency employees, elected officials, and many others, who are dedicated to bringing promising therapies to all people facing this devastating disease. 

It is only together that we will defeat ALS. 

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