Therapeutic developments in spinal muscular atrophy
نویسندگان
چکیده
منابع مشابه
Therapeutic developments in spinal muscular atrophy.
Spinal muscular atrophy (SMA), a potentially devastating disease marked by progressive weakness and muscle atrophy resulting from the dysfunction and loss of motor neurons of the spinal cord, has emerged in recent years as an attractive target for therapeutic intervention. Caused by a homozygous mutation to the Survival of Motor Neurons 1 (SMN1) gene on chromosome 5q, the severity of the clinic...
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ژورنال
عنوان ژورنال: Therapeutic Advances in Neurological Disorders
سال: 2010
ISSN: 1756-2864,1756-2864
DOI: 10.1177/1756285610369026