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Weight and fat gain may eventually lead to metabolic syndrome and type 2 diabetes, manifested by insulin resistance, glucose intolerance, dyslipidemia and hypertension, and an increased risk for cardiovascular illness [12,13,14]

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Studies have shown that Pip6a-PMO effectively rescues the disease phenotype and improves the survival rate of severe SMA mice, showing strong efficacy in the central nervous system and peripheral tissues