spinal muscular atrophy


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spinal muscular atrophy

n.
Any of several forms of a hereditary, progressive disease that involves loss of motor neurons in the spinal cord and brainstem and is characterized by muscle weakness and atrophy that vary in severity and age of onset.
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References in periodicals archive ?
NASDAQ: IONS) to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA), the company said.
Kristen Resendez, mother to a child with spinal muscular atrophy, on being hopeful a treatment newly approved by the FDA will bolster Texas to start screening all babies for spinal muscular atrophy.
Compound heterozygous mutation in two unrelated cases of Chinese spinal muscular atrophy patients.
Spinal Muscular Atrophy (SMA) is characterizsd by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.
There is currently no approved therapy in the EU for the treatment of spinal muscular atrophy.
The report provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
Conclusion: Among the floppy babies that were referred to the electro-diagnostic department at AFIRM for electro-diagnostic evaluation, spinal muscular atrophy (SMA) was the commonest electro-diagnostic impression.
The clinical trial into a new drug for Spinal Muscular Atrophy Type 2 will no longer take place at Newcastle
Spinal Muscular Atrophy Support UK was originally known as the Jennifer Trust in memory of Wellesbourne sufferer Jennifer Macaulay, who died in 1985.
Lefebvre S, Burglen L, Reboullet S, Clermont O, Burlet P, Viollet L; identification and characterization of spinal muscular atrophy determining gene cell 1995;80:155-65.
Spinal muscular atrophy is a devastating hereditary disease and is the biggest genetic killer in infancy.
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by lower motor neuron loss and progressive muscle weakness.

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