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Global Genes™ Allies in Rare Disease
2016 Overview
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RARE Webinar: Collaboration Among
Foundations
July 11, 2018
Thank you to our sponsors
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Collaboration Among Foundations
To avoid any technical difficulties and to listen in without any problems, please login through WebEx first before calling in.
You may be able to call in through your phone or computer. The number to use to call in is to the right of this screen.
Use this Access Code to join: 667 703 465
And your individual ID number was emailed to you.
If you have any technical issues or concerns, please reach out to Ashley, [email protected].
US TOLLFREE+1-855-749-4750US TOLL+1-415-655-0001
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Throughout the webinar, panelists will be taking questions from the audience
To send in questions, please use WebEx’s Q&A feature in the bottom right hand corner
Please feel free to submit questions throughout the presentation
Submit Your Questions
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RARE Webinar: Collaboration Among
Foundations
July 11, 2018
Thank you to our sponsors
Meet Today’s Panelists:
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Daniel LevineLevine Media Group
Kari RosbeckPresident and CEO
Tuberous Sclerosis Alliance
Kelly RanalloFounder and President
Turner Syndrome Global Alliance
Moderator:
Panelists:
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Kari RosbeckPresident and CEO
Tuberous Sclerosis Alliance
The Importance of Collaboration in Fulfilling Your Mission
Kari Luther Rosbeck, President and CEO
Slide 8
The TS Alliance, founded in 1974, is committed to finding a cure for tuberous sclerosis complex while improving the lives of those affected:
by developing programs,support services and resourceinformation;
by stimulating and sponsoringresearch; and
by creating and implementing public and professional education programs designed to heighten awareness of the disease.
www.tsalliance.org
Tuberous Sclerosis Alliance
Slide 9
Tuberous sclerosis complex (or TSC) is a rare genetic disorder that causes tumors to form in vital organs, primarily the brain, eyes, heart, kidney, liver, lungs and skin.
TSC affects ~1 in 6,000 live births and 1/3 of infants with TSC will develop infantile spasms
An estimated 50,000 Americans have TSC, and more than 1 million worldwide.
No two people are affected the same way, not even identical twins.
Neurological manifestations are often the most devastating – affecting almost all with TSC in some manner, from mild to severe.
TSC is the leading genetic cause of autism and epilepsy and leading symptomatic cause of infantile spasms.
About Tuberous Sclerosis Complex
Creating Successful Collaboratives for Awareness
Slide 10
Collaborating to Improve Patient and Transition Care
Slide 12
Partnering to Accelerate Research and Patient Support
Five Regional TSC and LAM Conferences, 2016-2017
Externally-Led Patient-Focused Drug Development Meeting on TSC and LAM, June 21, 2017
2018 Research Conference, June 22-24, 2017
Co-funding Research Grants
Encouraging Drug Discovery through Preclinical Consortium
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Kelly RanalloFounder and President
Turner Syndrome Global Alliance
Kelly Ranallo – Founder/President
We all have a story…
And every story has a
beginning
What is Turner syndrome?
Turner syndrome (TS) is a chromosomal disorder characterized by a complete or partial absence of the second sex chromosome.
TS affects one in 2,000 females. There are approximately 80,000 women, girls and babies with TS in the United States.
Typical karyotype – 46XXTS karyotype – 45XO or partial deletion of second X
chromosome
Creating a New Model of Care: Healthcare/Patient Collaboration
2011 2018
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TS Clinics Locations
Securing the future
TS Advocacy Organizations
BRAZIL
JAPAN
Kansas City – 2015 World Series
Unmet Rare Needs
Our Mission
RareKC will transform the
understanding, diagnosis and
treatment for rare disease in Kansas
City .
The passion of one…can transform the future of millions
Thank you!!!
www.RareKC.org
Questions and Answers
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Daniel LevineLevine Media Group
Kari RosbeckPresident and CEO
Tuberous Sclerosis Alliance
Kelly RanalloFounder and President
Turner Syndrome Global Alliance
Moderator:
Panelists:
Thank You Panelists :
Join us October 3-4, 2018 at our RARE Patient Advocacy Summit
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Registration Now Open!
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