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The New England Journal of Medicine, ISSN 0028-4793, 11/2017, Volume 377, Issue 18, pp. 1713 - 1722
Journal Article
Lancet Neurology, The, ISSN 1474-4422, 2017, Volume 16, Issue 4, pp. 264 - 264
  Several topics discussed at the stakeholder meeting were the same as those identified in the Duchenne muscular dystrophy discussion, including variability of... 
Neurology | CLINICAL NEUROLOGY | Medical colleges | Ophthalmology | Patents | Stakeholders | Collaboration | Muscular dystrophy
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 02/2018, Volume 378, Issue 5, pp. 487 - 488
To the Editor: In two reports regarding patients with type 1 spinal muscular atrophy (Nov. 2 issue), 1,2 researchers describe therapies involving antisense... 
MEDICINE, GENERAL & INTERNAL | Muscular Atrophy, Spinal | Spinal Muscular Atrophies of Childhood | Humans
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 02/2018, Volume 378, Issue 5, p. 487
Antibodies to scAAV9 could present a barrier to repeated administration.[...]we are curious to know whether infants who were treated with scAAV9 were tested... 
Studies | Proteins | Medical research | Immunoglobulins | Gene therapy | Gene expression | Vectors (Biology) | Children & youth | Spinal muscular atrophy
Journal Article
Drugs, ISSN 0012-6667, 3/2018, Volume 78, Issue 3, pp. 293 - 305
Journal Article
Journal Article
Neuron, ISSN 0896-6273, 01/2017, Volume 93, Issue 1, pp. 3 - 5
Spinal muscular atrophy is the most common genetic killer of infants. A therapy shows promise in the clinic, but there is a potential limit to its efficacy. In... 
spinal muscular atrophy | lncRNA | SMN | SMA | antisense | RESCUE | RNA | ANTISENSE OLIGONUCLEOTIDES | NEUROSCIENCES | MOUSE MODEL | Genetic Therapy | Muscular Atrophy, Spinal - genetics | Humans | Spinal muscular atrophy | Medical colleges | Atrophy, Muscular | Neurosciences | Proteins | Disease | Neurons | Rodents | Clinical trials | Gene therapy | Gene expression | Drug dosages
Journal Article
Journal Article