York / Piscataway / Carlsbad / Philadelphia / Salt Lake City / Boston / New York / New Orleans / Cambridge / Millville / Long Island / /
Company
Novartis / Roche / GlaxoSmithKline / Sanofi / Weston / Boston’s Paratek Pharmaceuticals / Nature America Inc. / Isis Pharmaceuticals / Biogen Idec / Genzyme / Harbor Laboratory / Merck SMN / Investigators Network / Nature Medicine / SMA Massachusetts / ISIS Pharmaceuticals Inc. / /
Country
United Kingdom / /
Currency
USD / /
Event
Product Issues / FDA Phase / Person Communication and Meetings / /
Facility
National Institute of Neurological / National Institute / Nationwide Children’s Hospital / Children’s Hospital / Spring Harbor Laboratory / University of Paratek Pharmaceuticals Small / Ohio State University / University School / Rapunzel’s tower / Boston Children’s Hospital / University Project / /
IndustryTerm
machinery / drug giant / drug giants / nervous systems / comparable gene therapy products / pharmaceutical / /
Institut National / Utah School of Medicine / University School of Medicine / SMA Foundation / US National Institute of HDAC / Columbia University Medical Center / office of Translational Research / DNA / Nationwide Children’s Hospital in Columbus / Academy of Neurology / Ohio State University in Columbus / steady-state metabolism Academy / Généthon / French Muscular Dystrophy Association / Boston Children’s Hospital / Center for Gene Therapy / National Institute of Neurological / Center for Gene Therapy at Nationwide Children’s Hospital / /
Person
Frank Bennett / Claudia Chiriboga / Brian Kaspar / Marina Corral / Nat / Luca Santarelli / Preclinical Dreyfuss / Arthur Burghes / Elie Dolgin / Kathryn Swoboda / Vivienne / Jerry Mendell / James Rusche / Karen Chen / Elliot Androphy / Jill Jarecki / Helena / Charles Camarda / Basil Darras / Helge / Lara / Adrian Krainer / /
Position
head / biologist / chief scientific officer / news editor / drug As director / study author / director of the pediatric Stroke / Fellow trial site director / ED messenger / messenger / head of research / site coordinator / research director of Families / director / head of neuroscience / biochemist / expression and pediatric neurologist / molecular biologist / /
Product
valproic acid / SMNRx / copy / gene / /
ProvinceOrState
Ohio / New York / Utah / California / New Jersey / Maryland / Illinois / P.N. / Massachusetts / /
npg Call in the backup The most common genetic killer of infants, a disease known as spinal muscular atrophy, is caused by mutations in a single gene. The human genome contains its own backup system—near-identical cop