The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: results from trials in healthy adults and patients with spinal muscular atrophy

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Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes muscle atrophy and weakness and devastating disease-related complications. It is the leading genetic cause of mortality in infants, with an incidence of 1 in 6,000−11,000 live births and a carrier frequency estimated at 1 in 40−60 [1-3]. SMA has a broad range of severity and a heterogeneous patient population, and is categorized into Types 1 through 4, defined by age of onset and the most advanced motor milestone achieved.

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