Former U.S. Congressman Dr. Phil Gingrey and Senior Advisor to the District Policy Group at Drinker Biddle has published a column in The Hill focused on the need for earlier detection and treatment of Spinal Muscular Atrophy (SMA) in infants.

Dr. Gingrey explains that SMA is the number one genetic cause of death for infants and approximately 10,000 to 12,500 people in the U.S. are living with the disease. SMA is caused by a mutation in a motor neuron gene, which causes nerves that control muscle function to stop working and eventually die. While children with SMA still grow cognitively, their bodies continue to deteriorate over time.

As a former OB/GYN for 26 years, Dr. Gingrey shares his concern that SMA is not currently a routine part of newborn screenings. Because symptoms of SMA are similar to some other diseases, signs of developmental problems are often dismissed, missed or misdiagnosed. 

While there is a simple blood test that can detect more than 95 percent of SMA cases, the Federal Secretary's Advisory Committee on Heritable Disorders in Newborns and Children has not reviewed SMA screening since 2008. Since then, several promising drugs that can potentially treat SMA have been developed and there are now six programs in clinical trials, including one that will most likely be submitted to the Food and Drug Administration for approval later this year.

“We’re at the doorsteps of a solution to treat this terrible disease. The faster we can get an FDA-approved drug on the market, the faster we can give thousands of children a chance to lead healthy and productive lives,” explains Dr. Gingrey. “August is SMA Awareness Month. What a unique privilege it would be to honor those affected by SMA by taking this important step.”

Click here to read Phil’s full column in The Hill.
Source: The Hill
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