US Tax Deductible EIN: 26-0256621
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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.
Keep up to date with the three leading research organizations:
Sio Gene Therapies: www.siogtx.com
Taysha Gene Therapies: www.tayshaGTX.com
The CTSF is very pleased to announce our latest grant in support of future clinical trials for Tay-Sachs and Sandhoff disease. This is your donation money at work!!
The CTSF proudly awards the labs of Queens University in Ontario, Canada $154,660 USD. This money will support and pay for the already initiated work to best understand the methodology and volume of Vector (medicine) to administer to children Intrathecally. This work is part of the complicated journey leading to the safe administration of gene therapy to children.
We will keep you posted on the progress on this work right here.
Donations to the CTSF are fully tax deductible and can be made in any amount by credit card at: www.curetay-sachs.org
Thank you for your continuing interest in FINDING THE CURE!!!!!
Dear Tay Sachs and Sandhoff patient communities,
On behalf of Sio Gene Therapies, it is our privilege to share an exciting update. Today, we are delighted to announce that the first patient has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff Disease.
The two-part AXO-AAV-GM2 study consists of (1) a dose ranging cohort evaluating the safety and efficacy of various doses of the gene therapy, followed by (2) a confirmatory cohort treated with the optimal dose identified from the dose ranging cohort. Both infantile and juvenile patients with GM2 gangliosidosis will be enrolled in the study which is being conducted in partnership between the University of Massachusetts Medical School and Massachusetts General Hospital (MGH)under the direction of Dr. Terence Flotte and Dr. Florian Eichler respectively, who work together to provide the treatment and follow-up of the patients enrolled in the study.
Information about the study, including inclusion/exclusion criteria, are posted on clinicaltrials.gov (NCT04669535): A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease - Full Text View - ClinicalTrials.gov. As with all clinical trials, enrollment is dependent upon the study investigator's medical assessment of the patient meeting the inclusion criteria for the study, and as such, Sio will play no role in the selection of patients for the trial. A few of the criteria are listed below:
As always, your treating physician will serve as the best source of information for ongoing medical treatment and management of your child's disease. To learn more about this study, you or your doctor may contact the Clinical Research Coordinator at MGH, Haley Andonian(firstname.lastname@example.org).
This is an important milestone for the entire GM2 community made possible by the many families, researchers and patient organizations committed to finding new treatments for Tay-Sachs and Sandhoff disease, and we look forward to enrollment of additional patients in this program. If you would like to reach out directly with any questions or feedback, you can contact us at email@example.com.
Gavin Corcoran, MD
Chief R&D Officer
Erika De Boever
VP, Clinical Development
Since our creation in June 2007, the CTSF has raised $4,995,464 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!
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.
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Alyssa Tyner was born on a pretty morning on June 19, 2014. We were so happy and blessed to finally have our family complete. Alyssa was the little sister to Leilani, who felt proud to be the big loving sister.
Krystie Anna Karl-Steiger was born on January 26, 2006 on a warm sunny winter day in California. Our precious little girl had a full head of hair, and gulped down her first bottle in record time.
Ryan Anthony Babo was born February 19, 2002. Like all Tay-Sachs children, he was absolutely beautiful. I remember lying in bed next to him when he was just a week old, watching him sleep, wondering what I had ever done in my life to deserve him.
Molly Grace was born on September 26, 2003. She was born 2 months early and weighed only 4lbs 0oz. Although she was so small she was very healthy.
Elise Catherine ten Berge was born on February 5, 2003. She was our first child and as all new parents do, we thought about what Elise's future would be like: her first word, her first step, and her first day of school.
Dakota Jean Bihn was born on May 30, 1999. She was a beautiful baby, weighing in at exactly 8 pounds. Everything about her seemed perfect. During Dakota's first year, she developed as a normal baby should. She sat up, rolled over, ate, and walked, all when she should.
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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On March 16, 2007, Isaiah came into the world at 9:41 PM weighing 8 lbs. 6 oz. he was greeted by his daddy, Grandma Lisa, and Great Aunt Chaundra.
Rachaeli entered this world with her eyes open wide; God delivered her to us shortly before the start of the Sabbath on Friday October 25, 2002. It was a perfect delivery, following a perfect pregnancy. Rachaeli was absolutely captivating.
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Travis Cole Snellgrove was born to Wendi and Travis on April 12th, 2004. He was born with no problems and was home the next day. He was a very good baby and we had no problems with him for the first month, then he started having trouble.....
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Amelia was born September 29, 2006 and was a sweet, easy-going baby right from the start. Her baby book filled up as it should have first smile at four weeks, rolled over at four months, babbled at five months.
Charles Lee Anderson was born on February 26, 2006 to Bryce and Amy Anderson. His sister Anna was 14 and brother Ben was 11. Charles Lee was so perfect.
Emma Rose was born on May 17, 2004. She was very eager to enter our world - arriving before my doctor could even get to the hospital! She was a beautiful, healthy, and sweet baby.
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Mallory Rayne Salazar is a ray of light that came to us one rainy Saturday afternoon. She was born on July 14, 2007 and was 7lbs. 8oz. of pure perfection. From the first time I laid eyes on her, I knew there was something "special" about her. Little did we know just how much she would teach us.
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The Cure Tay-Sachs Foundation is exempt under Section 501(c)(3) of the Internal Revenue Code, making this gift tax deductible to the fullest extend permitted by law.
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