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Current Focus

Items that require ACTION. On this page you will find information on any topics and issues that we, the Spinal Muscular Atrophy community, need to be aware of, and often, take action.


Check back often, as we will be updating this page frequently.


Sholar Rock - Topaz Trial

Pharmaceutical company Scholar Rock is finishing phase II of their TOPAZ clinical trial, a study of a drug they are calling SRK-015 which inhibits the production of Myostatin, a protein found to inhibit skeletal muscle growth.

Click here to find our more about this trial, our advocacy efforts to include adults over the age of 21 and type 1 in their trial, and how you can HELP.


RISDIPLAM - Genentech

Risdiplam (developed in collaboration with the SMA Foundation and PTC Therapeutics) is a small molecule used to increase the amount of SMN protein made by the SMN2 gene, commonly referred to as back up genes.

Click here to learn more about this treatment.

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