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Therefore, while encouraging, particularly in ameliorating certain PD symptoms, its efficacy and safety must be confirmed through larger, well-designed clinical trials

Spinal Muscular Atrophy (SMA) Spinal Muscular Atrophy Statistics : It is generally believed that SMA is currently present in 10,000 to 25,000 children and adults in the United States and is the leading genetic cause of death to infants and toddlers

Browning, Leanne M et al