Project Rare is a documentary film that tells a remarkable story of hope and struggles against rare neurological diseases in children. Funded by the B Brave Foundation, the film will raise awareness of and support funding for research that leads to cures.
Joey Scaparotti was diagnosed with a rare neurological disorder when he was just 15 months old. After learning about his progressive and incurable infantile Gangliosidosis-1, or GM1, Joey’s parents Sam and Sara devoted themselves to giving Joey the best care possible at home. Joey lost his battle at just three years of age, but he lives on through a movement that is changing the lives of children and families facing rare fatal neurological disorders. The B Brave Foundation enhances the quality of life for children and adolescents affected by these rare ailments by supporting them and their families, empowering caregivers, and ultimately pursuing treatments and cures.
Closely related to Tay-Sachs disease, GM1 is a lysosomal storage disorder that is caused by the absence of a vital enzyme called beta-galactosidase (GLB1) due to an inherited gene mutation. Because of the absence of GLB1, gangliosides cannot be broken down in the brain, and causes it to accumulate to toxic and fatal levels.
Lysosomal storage disorders have no cures or effective treatments. They are rare and therefore research is underfunded compared to other diseases. Through B Brave and other efforts, that is starting to change.
The University of Massachusetts Medical School is collaborating with Axovant Sciences to help fund early clinical trials for investigational gene therapies for GM1 Gangliosidosis, Tay-Sachs and Sandhoff diseases.
Project Rare will raise awareness of these diseases, in turn helping fund promising new research into treatments and cures. Until cures are found, B Brave also provides families struggling with a similar diagnosis with comfort, support and hope. The documentary also offers tools and resources to help family, friends and caregivers of those affected to more effectively help and comfort those affected.
Your donations will pay for the production of the documentary. Any additional funds will support B Brave’s mission.
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