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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In May, the FDA had given approval for the use of Zolgensma for use by spinal muscular atrophy patients less than two years old.
(Catalyst) (Nasdaq: CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced its support of Spinal Muscular Atrophy (SMA) Awareness Month.
Food and Drug Administration in May -- out of reach for many parents of infants with spinal muscular atrophy.
The news was met with joy by those such as Heidi Prescott-Booth, 11, from Wolverhampton, who also has spinal muscular atrophy and whose family have previously told of her relief at the decision.
According to MDA, Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of paediatric patients less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, including pre-symptomatic at diagnosis.
It seems that when it comes to treatment of spinal muscular atrophy, the earlier the better.
The positive recommendation is for the treatment of infants, children and adults with 5q spinal muscular atrophy (SMA), including pre-symptomatic and symptomatic SMA Types 1, 2 and 3.
Sam McKie, aged six, has spinal muscular atrophy and father Gary believes his life could be transformed by a drug called Spinraza.
Sam McKie, six, has spinal muscular atrophy and his dad Gary believes his life could be transformed by a drug called Spinraza.
"Will to fly" portrays a family's life with a daughter who has type II spinal muscular atrophy (SMA), a regressive genetic disease that increasingly affects a person's ability to function.
[USA], July 17 (ANI): A new study suggests that children with Spinal muscular atrophy (SMA) can achieve improvements in motor function after six months of treatment with the drug nusinersen, particularly when treatment began before seven months of age.

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