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Genetic genealogy / Spinal muscular atrophy / SMN1 / Survival of motor neuron / Antisense therapy / Amyotrophic lateral sclerosis / SMN2 / Sense / Gideon Dreyfuss / Motor neurone disease / Biology / Genetics


Spinal Muscular Atrophy & ISIS-SMNRx Isis Pharmaceuticals is developing a drug to treat Spinal Muscular Atrophy (SMA). SMA is a genetic neuromuscular disease characterized by muscle atrophy and weakness. SMA is a leading
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Document Date: 2013-11-30 18:15:27

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Company

Spinal Muscular Atrophy & ISIS-SMNRx Isis Pharmaceuticals / ISIS Pharmaceuticals Inc. / /

Continent

Europe / /

Country

Japan / United States / /

Event

FDA Phase / /

MedicalCondition

cancer / diseases / Spinal Muscular Atrophy / disease / severe and rare diseases / genetic neuromuscular disease / cardiovascular disease / inflammatory / muscle atrophy / /

OperatingSystem

ISIS / /

Organization

Muscular Dystrophy Association / SMA Foundation / /

Position

leader / /

Product

Hammersmith Functional Motor Scale-Expanded / ISIS-SMNRx drug / /

Technology

pharmacokinetics / drug discovery / human genome / /

URL

www.isispharm.com / http /

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