US Tax Deductible EIN: 26-0256621

- (562) 212-1071

- 2409 E. Luke Avenue Phoenix AZ 85016

US Tax Deductible EIN: 26-0256621

- (562) 212-1071

- 2409 E. Luke Avenue Phoenix AZ 85016

FIRST TREATMENT REPORT!!! A MUST READ!!!

Dear Rick -

Earlier this morning, Axovant, in partnership with our collaborators at U Mass, reported three-month data from an investigator-initiated study administering investigational AXO-AAV-GM2 gene therapy in a patient with advanced infantile Tay-Sachs disease. Additional details about this data can be found here:

AXOVANT ANNOUNCES CLINICAL UPDATE FROM FIRST TAY-SACHS DISEASE PATIENT DOSED WITH AXO-AAV-GM2 GENE THERAPY

As Gavin mentions in the press release, this is the first time a gene therapy has been administered to a child with Tay-Sachs disease, and we are encouraged by these early clinical results, which will help inform the design of our future clinical study for AXO-AAV-GM2.

Please take a look at the information in the release, and should you have any questions, we would be happy to discuss further with you.

Regards,

Parag V. Meswani, PharmD

SVP, Commercial Strategy & Operations

Axovant Sciences, Inc.

Our Mission

The Cure Tay-Sachs Foundation is dedicated to funding the on-going research needed to find treatments and a cure for Tay-Sachs Disease.

Once the cure for Tay-Sachs has been found - and we will find it! - we will dedicate our efforts to promoting carrier-testing and Tay-Sachs awareness initiatives.

To begin the journey into Tay-Sachs Awareness, click here.

What is a Clinical Trial???

Cure Tay-Sachs Foundation

The Cure Is Within Reach!

Since our creation in June 2007, the CTSF has raised $4,647,829 from thousands of kind and generous donors. We have issued research grants to 9 researchers/laboratories around the world, and made impressive progress towards our ultimate goal... The Cure!

There is HOPE in your HELP!!!

Donate Now

2019 Donor Recognition

Research PhD: $1,000 to $4,999 Donation - Bryce and Amy Anderson, Ervin Cohen and Jessup, FACEBOOK DONORS, Lauren Rabinowitz Rabinowitz *** Treatment Provider: $250 to $499 Donation - 4300 Jasmine Property, FACEBOOK DONORS, Karl and June Reidel, Lillian Ponce De Leon *** Symptom Manager: $101 to $249 Donation - Accenture, Adam Anderson, Amazon Smile, Amy Brown - InstaPhysique, Benevity, Dan Duggan, Gloria Nelson, Haprinder Singh, Julie Rabinowitz, Kimyetta Barron, Lisa Nelson Laptalo, DMD, Megan Lehnhoff, Network For Good, Phillip Starr, Raquel Richarte, Roxie Food Center , Tami Kint, United Way - Loraine *** Jacob Sheep Parent: $100 Donation - Andrea Barnes Trust, Carlene Klein, Charles Barrington, Cynthia Miller, James & Heather Scullin, Jewish Federation of Atlanta, John Daise, Joseph Vumbacco, June and Mike Martz, Matthew Marcus, Nan Chapman, Patricia Beaudry *** Care Provider: $1 to $99 Donation - Audrey Ades, Bank of America, Brad Devine, Crystal Bledsoe, Deborah Marnien, Eduardo Vazquez, Elizabeth Masiero, Estefania Becerra, Filippo Moreci, Fuquay-Varina Garden Club, Hal and Joni Krevoy, Janet Schmidt, Jerald & Jaclyn Baranoff, John Marnien, JonMarnien, Larry and Aljean Webb, Marina Mochkina, Michael O'Rourke, Pledgeling Foundation, Richard & Julianne Schuck, Ruth Goldberg, Ruth Rosenfeld , Sagar Mehta, Sarah Mattingly, Susan McGuire, Tara Keefner, Teresa Castelli, Terri Yursky, Thurs Morning Coffee League, United Way CCR, Vish Nadaarajah, Yolanda Thomas, Your Cause, Your Cause HCA *** Donations made in honor of: - Charles Lee, Dakota, Davita Schachter, Dillon, Emma, Erin Merrien, Gillian Goldberg, Grayson K, Grayson L, Hike for Grayson!!, Krystie, Mallory S., Natalie Barnes, Nathan R, Rosea Comer, United Way - Sacramento ***

Click here to view Donor List

Donate In Honor or Memory Of: (click on button(s))

In Honor or Memory Of

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Tay-Sachs disease is caused by the absence or insufficient level of a vital enzyme called Hexosaminidase A (Hex-A). Without Hex-A, a fatty substance or lipid called GM2 ganglioside accumulates abnormally in cells, especially in the nerve cells of the brain. This ongoing accumulation, also called substrate, causes progressive damage to the cells. In Classic Infantile the destructive process begins in the fetus early in pregnancy, although the disease is not clinically apparent (symptoms do not start) until the child is several months old. By the time a child with Tay-Sachs disease is three or four-years old, the nervous system is so badly affected that life itself cannot be supported. Even with the best of care, all children with classic Tay-Sachs disease die early in childhood, usually by the age of 5, although some do live longer.

How do set up a free Facebook Fundraiser?

Many of you have asked how to set up a FB Fundraiser.... here ya go. You can create fundraisers for charitable organizations and personal causes on Facebook for Birthdays, Anniversary's, or any occasion!

To create a fundraiser for the CTSF: (no cost to you!)

1.  Click Fundraisers in the left menu of your News Feed

2.  Click Raise Money...

3.  Select Nonprofit/Charity (Cure Tay-Sachs Foundation)

4.  Select a charitable organization, choose a cover photo and fill in the fundraiser details

5.  Click Create

IT'S FREE, IT'S FUN, AND ALL $$$ GOES TOWARDS RESEARCH

2018 Year End Summary

The work that we did in 2018 brought us to exciting new possibilities for the future. It's hard to say we had a "good" year when we lost kids and more kids were diagnosed. Sometimes it just seems like "new kids just keep coming" every few weeks. But 2018 did bring some hope for both todays kids and future kids who are burdened with this horrible disease. The cause continues and the fight endures for all our kids.

Here are some of the highlights:

  • Eleven years ago your donations funded the early work at the University of Massachusetts Medical School for research. In November of 2007, we sent them $50,000. By January of 2009, that number swelled to $300,000. Much more funding was given from us and other partners , and the rest as they say, is history. Axovant now owns much of the intellectual property generated, and clinical trials on real kids are hopefully right around the corner.
  • IntraBio hopes to trial their IB1000 series of drugs here in the US in 2019. More details to follow as they seek regulatory approval
  • We had our best financial year since 2014 with $355,412 donated!! We had 51 Fundraising efforts, both large and small, and 1811 donations. That's an average of 5 donations per day, everyday. We have a war chest that will enable us to be nimble and to fund the most promising and compassionate investments that present themselves in the future.

So, even though we are dealing with a really crappy disease, we really did have a very good year. We have more hope and more determination than ever to find an end to these diseases. And, for once, the light at the end of the tunnel doesn't feel like the headlamp of an approaching train.

THANK YOU all for your enthusiasm, your support, your donations, and your interest in our kids. On behalf of myself and our entire Board of Directors, we will continue to work hard not to let you down.

- Rick and the CTSF Board

Tanganil: An easy way to share your experience...

Cure Tay-Sachs Foundation (CTSF) and NTSAD have collaborated with TREND Community to collect information from families and individuals about their experiences using Tanganil, available without a prescription from Europe and indicated for the treatment of positional vertigo (dizziness). It has not been proven to be effective or approved for use to treat symptoms of GM1 and/or GM2 Tay-Sachs and Sandhoff.

  • There is no travel required to take part in this online community.
  • All data shared will be anonymous and de-identified.
  • The data will be used by researchers, doctors, and pharmaceutical companies to better understand how the disease impacts the family, generate ideas for future research and design better clinical trials.

Click the link below to download the TREND Community PDF to read more about this initiative, how it can help and how you can sign up to be a part of this unique way to participate in research.

Here is the link. The instructions to sign up are right at the bottom.

http://news.trend.community/home/gm1-gm2-tanganil-health-initiative

Rick Karl, CTSF

Email: Rick.Karl@curetay-sachs.org

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Ryan's Story

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Conner's Story

Conner, a precious miracle and an amazing creature sent to Carl, Brenden and myself one cold, snowy January day. From the second he entered our life, I knew in my heart he was so incredibly special. His first smile, his first cry, even his first breath, he was--and remains--beautiful.

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