Texas is considering adding spinal muscular atrophy (SMA) to its list for newborn screening in the wake of the U.S. Food and Drug Administration’s approval in December of Spinraza (nusinersen), the first treatment approved for a broad range of SMA patients.
The addition of SMA to Texas’ newborn screening list may help people like Kristen Resendez, whose son, Jack, was diagnosed with SMA at seven weeks of age, Resendez told Alexis Aguirre in an article published in the Valley Morning Star.
Jack, who turns 2 this month, took part in a clinical trial testing Spinraza. She expects the boy will be on the therapy for most of his life.
Resendez traveled to the capitol in Austin recently to speak with the Texas Newborn Screening Advisory Committee in favor of statewide prescreening. Now that a treatment is available, Resendez thinks the advisory board should add SMA to the screening list, especially as a lack of a therapy was a major reason given for not testing.
Spinraza, administered by intrathecal injection, was developed by Ionis Pharmaceuticals and is marketed by Biogen.
The spinal muscular atrophy (SMA) support and advocacy organization Cure SMA is spearheading the newborn screening campaign. The nationwide effort was announced at the 2017 Annual SMA Conference held June 29-July 2 in Orlando, Florida.
Texas’ consideration of adding SMA to its newborn screening program comes on the heels the Missouri Legislature passing a bill that made it the first state to require screening of all newborns for SMA. That bill, sponsored by state Rep. Becky Ruth, was signed into law by Gov. Eric Greitens on July 10.
To build on its momentum in Missouri and Texas, CureSMA also has created a new chapter in Arkansas to support families and research efforts, as well as raise awareness about screening newborns for SMA.
CureSMA is soliciting everyone’s help to achieve nationwide newborn screening for SMA. Being a non-profit organization, donations play a significant role in how the organization funds initiatives and research to continue supporting and advocating for the community.
CureSMA accepts several types of donations, in addition to funds. The organization has an equipment pool where medical equipment can be loaned free of charge. The organization recently received a donation of 14 special tomato feeder seats and 80 large sheepskin blankets from the Bugenske family, who are the parents of Leo, a South Carolina boy who has SMA type 1 and have together founded Leo’s Pride.
The donation of the feeder seats helped Cure SMA fulfill the requests of each family on the equipment waiting list and the donation of large sheepskin blankets helps fund a new support program, in which Cure SMA is now able to offer these larger blankets to older children with SMA. Visit [email protected] to learn more about donations.