The development and subsequent physical and mental decline in people with neurodegenerative conditions such as Parkinson’s disease，amyotrophic lateral sclerosis（ALS）and Alzheimer’s disease have a devastating impact on the lives of those affected and their families。Pursuing innovation is critical to changing the course of these diseases and supporting those living with them and their caregivers。

SMA is arare，devastating genetic disease that leads to progressive muscle weakness，paralysis，and，when left untreated in its most severe forms，permanent ventilation or death in90%of children by age2.It is aleading genetic cause of infant death.All those diagnosed with SMA should be to able benetty from，not， through turning promising transformative medicines into proven treatments。

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