Wisconsin State Goodwill Ambassador

Jacob Smith, Age 13 Burlington, WI Home and Family Jacob lives with his mom Bobbi Ann, dad Kerry, and sister Jessica. Jacob’s dog Minnie is small enough to ride with him in his power wheelchair! Diagnosis Jake was diagnosed with Spinal Muscular Atrophy Type 2 at 14-months of age. Spinal muscular atrophy (SMA) is a . . .

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Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Type 2 and 3 SMA

Press Release: Continued increases observed in multiple measures of muscle function in SMA children treated with ISIS-SMN Rx Statement from MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D.: “We are excited to see that Isis Pharmaceuticals reported positive results from patients who were enrolled in their Phase 2 trial and who continued to receive the drug after the . . .

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News: Isis Pharmaceuticals Reports Data from Phase 2 Study in Infants with Spinal Muscular Atrophy

On June 11, Isis Pharmaceuticals, Inc. released an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA).  The data showed that infants treated with ISIS-SMNRx have shown continued increases in median event-free survival, positive results in muscle function tests, and attainment of important developmental . . .

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