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Advocacy Groups
…EURORDIS SMA Europe TREAT-NMD Argentina Expand Familias AME Argentina Brazil Expand Amigos Da Atrofia Muscular Espinhal Canada Expand Families of SMA Czech Republic Expand Kolpingova rodina SmeÄno France Expand Association…
Read MoreSMA Organizations Worldwide
…Expand EURORDIS SMA Europe TREAT-NMD Argentina Expand Familias AME Argentina Brazil Expand Amigos Da Atrofia Muscular Espinhal Canada Expand Cure SMA Canada Czech Republic Expand Kolpingova rodina SmeÄno France Expand…
Read MoreThe New York Times: “A Deadly Disease of Infants Attracts New Research Money,” by Anahad O’Connor
…therapy to reverse the course of this,” said Dr. Charles Schleien, Owen’s pediatrician at Children’s Hospital of New York-Presbyterian. “We just try to make the children comfortable and decide whether…
Read MoreNeurology Today: “Advocacy for Neurological Disease: The ‘Family Business’ No Family Wants”
…wrong with her daughter. In 1984, when Stacie was ten, her primary physician, Mary Coleman, MD, a neurologist and founder of the now defunct Children’s Brain Research Clinic in Washington,…
Read MoreFrequently Asked Questions
…One in every 6,000 to 10,000 children is born with the disease. SMA has generally been believed to affect as many as 10,000 to 25,000 children and adults in the…
Read MoreISIS PHARMACEUTICALS REPORTS DATA FROM ISIS-SMNRX PHASE 2 STUDY IN INFANTS WITH SPINAL MUSCULAR ATROPHY
…ISIS-SMNRx with a primary endpoint of event-free survival. The CHERISH study is a randomized, double-blind, sham-procedure controlled fifteen month study in approximately 120 non-ambulatory children with SMA. The study will…
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