First drug to treat spinal muscular atrophy gets FDA approval: Costly injectable shown to stall debilitating muscle-wasting effects of rare genetic disorder

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Bibliographische Detailangaben
Veröffentlicht in:	American journal of medical genetics. Part A 2017-04, Vol.173 (4), p.837-838
Format:	Artikel
Sprache:	eng
Schlagworte:	Child Drug Approval - economics Humans Muscular Atrophy, Spinal - drug therapy Muscular Atrophy, Spinal - economics Muscular Atrophy, Spinal - genetics Mutation Oligonucleotides - economics Oligonucleotides - therapeutic use Survival of Motor Neuron 1 Protein - genetics Survival of Motor Neuron 2 Protein - genetics United States United States Food and Drug Administration
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