Spinal Muscular Atrophy (SMA) is a rare, inheritable, neurodegenerative disease, one of the most common genetic causes of infant death. Fifteen Ukrainian children suffer from SMA and need a lifetime-lasting dose of gene therapy Zolgensma. Zolgensma by Novartis costs over $2M, it is the most expensive medicine to date. The war in Ukraine has halted all fundraising efforts. Help us save the lives of children from Ukraine!
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