UPDATE: CureSMA workgroup revises guidelines
CureSMA workgroup has revised their guidelines and is now recommending immediate treatment for SMA patients with 4 smn2 copies, as requested.
Earlier last month, SMA United for Treatment Options sent a letter to CureSMA with the following ask: that their workgroup revise the following statement which appears on both Spinraza and Zolgensma policies at United Healthcare insurance:
"Patients with four copies of SMN2 should be screened periodically for symptoms and referred to a geneticist to determine the exact number of SMN2 copies, but the working group recommends against immediate treatment with a disease modifying therapy.”
We wanted to be sure the FDA label for these two treatment drugs are being adhered to, and newborns are treated regardless of the number of SMN2 copies present, as soon as they are detected by new born screening.
United Healthcare policies still need to be updated to reflect this change but the path to treatment should be easier now.