Christine Waggoner’s Post

Thankfully, the bet did pay off and Inside Out 2 is the fastest animated feature to ever reach $1B (19 days). Sending my warmest congratulations to the entire crew, and studio, particularly those who worked so hard and were impacted by the recent layoffs. This article from Time outlines the high stakes and an in-depth interview with Pete Doctor: https://lnkd.in/geZgC8EH

Sharing again for people to share with interested Californians! We're aiming for at least 1000 signatures. URGENT request to help in our fight for support of rare disease research funding: SIGN and SHARE: https://lnkd.in/emvehK9t Thank you!

Dear Fellow Californians and Rare Disease Community, URGENT request to help in our fight for support of rare disease research funding: SIGN and SHARE: https://lnkd.in/emvehK9t My daughter Iris suffers from a rare disease called GM1 gangliosidosis and there is a general dearth of funding given that there are over 10,000 rare diseases. The California Institute for Regenerative Medicine (CIRM) has been a great support to rare diseases and medical research. This would not impact only Californians. There would be broad ripple effects throughout drug development, biotechs, and research. Above all, for the families like mine who are in need of hope, we need avenues for meaningful funding for projects for rare diseases. Please take a few minutes and sign on and share. Thanks.

Showing my age, but as of yesterday: 25 years at Pixar... Inside Out 2 is out! Certified Fresh on Rotten Tomatoes: 93% Critical Tomatometer and 95% Audience Score! https://lnkd.in/gVSQnZiV

Please go see Inside Out 2 this weekend! https://lnkd.in/gKFdkHuH

This is a momentous announcement for ultra-rare diseases, particularly, lysosomal diseases with CNS involvement such as Sanfilippo. GM1 gangliosidosis is also a lysosomal disease with CNS involvement. May others in biotech take note and be so bold to make the same demands for accelerated approval based on biomarkers. There is no question that GM1 gangliosidosis is caused by an excess of GM1 ganglioside and other substrates. We must boldly pursue a more compassionate path for those who are literally dying in wait for treatments. After years in patient advocacy, I'm utterly convinced that possible treatments already exist. This is not a scientific issue. It's mainly a societal one. #curegm1 #raredisease #genetherapy #drugdevelopment #patients #advocacy #hope #takeaction

Sharon King's quote below and this whole piece by Steve Usdin is food for thought. Why have we built systems that lack compassion and expanded access for people who are literally dying in wait? Sufferers of ultra-rare fatal diseases are willing to take risks for a chance at life when the alternative is certain death. “These are real people, pulled to the edge of hope, only to be pushed from the cliff.” -Sharon, King #curegm1 #raredisease #helpnow #nevergiveup

Every single day we are working to create positive change at the Cure GM1 Foundation. Our June newsletter is here: curegm1.org/news. The strength of our community for GM1 Day was truly inspiring.

Thank you to Carolina Sommer and the founders of the NW Rare Disease Coalition for creating this platform. There are people in every corner of the world suffering from "rare" diseases. Together, we can continue to push for greater awareness and progress.