"It is important to emphasize that the conditions that are included on the newborn screening panel all result in serious medical complications (e.g., developmental delay) and/or death if not recognized early," Howell told the subcommittee.
Tucson, AZ (PRWEB) September 27, 2013
Pediatrician and medical geneticist R. Rodney Howell, who chairs MDA's Board of Directors, testified Thursday before the Subcommittee on Children and Families of the U.S. Senate Committee on Health, Education, Labor & Pensions to advocate for passage of the Newborn Screening Saves Lives Reauthorization Act of 2013 (S. 1417).
Howell emphasized that:
- conditions that can be detected at birth for which there are emerging therapies should be given priority in the review process for inclusion in the list of recommended newborn screening tests;
- priority review will not reduce the scientific rigor of the review process; and
- newborn screening reduces the overall cost of illness (sometimes called "burden of disease") for the conditions involved.
"It is important to emphasize that the conditions that are included on the newborn screening panel all result in serious medical complications (e.g., developmental delay) and/or death if not recognized early," Howell told the subcommittee. "All children with these conditions benefit from early diagnosis and treatment."
Three disorders in MDA's program are or may become candidates for addition to the Recommended Uniform Screening Panel (RUSP.) Pompe disease was presented to the Advisory Committee for possible inclusion in the RUSP in January 2013, and the Advisory Committee has recommended that it be added to the RUSP. Spinal muscular atrophy (SMA) was nominated several years ago but not deemed ready for full review by the Advisory Committee; a pilot for SMA newborn screening is under way. In February 2013, scientific information about the feasibility of newborn screening for Duchenne muscular dystrophy (DMD) was presented to the Committee.
A video of the Sept. 26, 2013, Senate subcommittee hearing is available.
To support the Newborn Screening Saves Lives Act, take five minutes to contact your U.S. senator here: mda.org/advocacy/get-involved/legislative-alerts.
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement. Visit mda.org and follow us at facebook.com/MDAnational and @MDAnews.