» Some 15,000 Americans have SMA, or spinal muscular atrophy.
» About one in every 6,000 babies is born with it. Most have SMA Type I, which usually kills a baby within two years. There are less-severe degrees, including an adult-onset SMA.
» A child with SMA inherits the gene mutation from each parent, although the parents typically aren’t affected by the disorder. About one in 40 people carries the gene that causes it.
» Scientists are working with drug therapies to produce more of the protein and gene therapies to replace the faulty gene. But clinical trials of new drug therapies are at least a year away if not considerably more, said Dr. John Porter of the National Institute of Neurological Disorders and Stroke.
Source: Families of SMA (a network of families, doctors and scientists)
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