Shifting the trajectory of diseases with high unmet needs first requires learning what changes will make meaningful differences for those most impacted. Addressing the debilitating symptoms of PBH could mean the difference between performing your job and being forced to leave it. For people living with neurodegenerative diseases, slowing progression and extending overall survival could mean being there for a milestone moment, like a wedding anniversary or the birth of a child, or maintaining independence and enjoying life’s ordinary moments for longer.
Firsthand experiences of community members motivate our work. We hope that you too can draw inspiration from the stories below.