First ALS Patient Receives Isis-SOD1RX Antisense Treatment in Phase 1 Clinical Trial Isis Pharmaceuticals, Inc. has initiated a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig’s disease also known as familial amyotrophic lateral sclerosis (ALS).  Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1.  The ALS Association and the Muscular Dystrophy Association are providing funding for the development of ISIS-SOD1Rx. ISIS-SOD1Rx is an antisense drug designed to inhibit the production of SOD1.  Antisense techniques are used to deactivate disease-causing or undesirable genes so that they cannot produce harmful or unwanted proteins. "This therapy will be the first treatment for ALS that is specifically aimed at the target, SOD1, known to cause ALS. The development of new treatments for ALS is an extremely challenging and costly process.  The ALS Association has been pleased to co-fund this study and partner with Isis on this extremely promising treatment approach. It is only through the support of our generous donors that this type of advancement is made possible," commented Lucie Bruijn, Ph.D., Chief Scientist, The ALS Association.   Read full story

because you can... Join Us! >>

Tribute Funds to Honor and Remember. Find or Create a Fund>>

Make a general gift to our chapter. Your donation supports The ALS Association's mission. Donate Now >>

Sign up for our Monthly eNewsletter "Connections" * First Name: Required * Email: Required   If you respond and have not already registered, you will receive periodic updates and communications from The ALS Association.   Remember me. What's this? Spam Control Text:   Please leave this field empty