Updated: Feb 5, 2020
Our goal: Advocating for the inclusion of adults over the age of 21 and the entire type 1 population with Spinal Muscular Atrophy (SMA)
SMA United for Treatment Options is engaging Scholar Rock and CureSMA regarding their Nusinersen/SRK-015 (TOPAZ) study.
We are thrilled about this development but concerned with the current age limit on type 2 and 3 and exclusion of type 1 patients with Spinal Muscular Atrophy.
In order for the drug to be approved and paid for all, we need data. In order to have data, we need to have adults type 2 & 3 in the study NOW and we need a plan for including type 1 patients.
We are seeking answers and suggesting ways to address this issue. The study is about to wrap up phase 2 and determine next steps - this is the time to intervene and inform further development!
Preliminary information shows great results on efficacy and safety for type 2 and 3 children and teenagers currently in the study.
AGAIN: OUR GOAL IS TO ENSURE ACCESS TO TREATMENTS FOR ALL!