The first FDA approval of a therapy for SMA has created an opportunity for our community to move forward on another of our long-term priorities: newborn screening. SMA is the leading genetic cause of death for infants, but we have an historic opportunity to change that through newborn screening and early treatment.
Currently, the average infant with SMA type I is not diagnosed until five months of age. This means that these babies are not receiving treatment during the critical early weeks and months of life, when research suggests it may be most effective.
In February, the federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) recommended that newborn screening for SMA be implemented nationwide. This recommendation was subsequently approved by Health and Human Services Secretary Alex Azar in July. Cure SMA was there at every step in the process, submitting the nomination and supporting it from acceptance to vote.
This is an important first victory as we work toward our goal of having every baby born in the United States screened for SMA, but much remains to be done. Each state must decide individually whether to implement SMA newborn screening.
Seven states—Illinois, Indiana, Minnesota, Missouri, New York, Utah and Ohio—have already adopted this recommendation. Three others—Georgia, Massachusetts and North Carolina—have begun pilot screening, and at least a dozen more are currently considering action.
Congress is currently in the process of determining the funding levels for two critical programs that help states develop new tests, meet scientific quality standards, and educate the public, along with healthcare providers, about newborn screening programs.
We need your help to make sure the federal government knows how important newborn screening is to ensure the best possible treatment for SMA. Take action today and tell your Senators and Representatives to support the highest level of funding possible for federal newborn screening programs.
Be sure to check out our news section later this week to learn more about Cure SMA’s approach to newborn screening!