Cafe Med: Seminar 4: Spinal Muscular Atrophy - A treatable childhood motor neuron disease
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10 years ago, Spinal Muscular Atrophy was the second most common inherited cause of infant mortality worldwide, but you may never have heard of it. Severely affected children had a life expectancy of less than 3 years, but now three novel and expensive therapies have emerged, which are providing new...