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The Cure Tay-Sachs Foundation is the leading private funder of Tay-Sachs research committed to finding therapies and a cure for this 100% fatal and heinous disease. Many of us know the personal heartache of losing our children. Sadly, our kids never had a chance. We have been working to change that.
I am delighted to share that a clinical trial request has been submitted to the FDA for a new, potential treatment for Tay-Sachs disease. Known as IB1001, this type of drug has been used in compassionate-use studies in Europe for many years to treat a number of neurogenerative diseases and lysosomal storage disorders like Tay-Sachs, and has been demonstrated to be very safe, well-tolerated, and most of all, effective.
As you all know, a US clinical study with IB1001 would be this community's first opportunity to intervene in the quick decline of our Tay-Sachs kids. Finally, perhaps, a breakthrough for a disease where we have nothing available. This gives us hope!!
The FDA does a magnificent job of bringing oversight and cadence to the process of approving clinical trials for new drugs like IB1001 that have the potential to help so many of our patients and our families. However, since a majority of our kids die so young, it will be unethical to apply the typical "before and after" benchmarks to determine IB1001's effectiveness. Collectively, I know we all believe that the FDA will find a better way to assess the effectiveness of IB1001 given the "death in infancy" nature of our disease, and help give our kids their first treatment option as soon as possible. This gives us hope!!
I have reached out the management team of the company (IntraBio) sponsoring this clinical trial. They have confirmed that based the safety and efficacy observed in previous studies, they intend to conduct a clinical trial with IB1001 for the treatment of Tay-Sachs patients in Europe, and hope to be able to open the study to US patients. At this time, they could not comment further on the details of their interactions with the FDA.
As we all know, "Time is Brain" with Tay-Sachs. We as a community of 3000 strong are excited, motivated and ready to work with the FDA to get this trial approved expeditiously!!
Please send questions, comments or thoughts to: Rick.Karl@curetay-sachs.org
Since our creation in June 2007, the CTSF has raised $4,429,639 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.
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!
5. Click Create
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.
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.
Rick Karl, CTSF
"Holy Cow! Over 2000 people "Like" the CTSF Facebook page now, and our this website has been visited 6000 times in the last 12 months. I'm humbled! It's March 5th, and we have already brought in over $100,000 this year for research. With what we have planned for the rest of the year, you ain't seen nuttin yet! Of course, we plan to write checks for over $800,000 this year. Our funding will see the first Gene Therapy treatment this year, and two other clinical trials on affected children! There will be obstacles, but there has never been..... not even close... this much hope. ALL our hope has been from your help. So, if you have a rich crazy Uncle someplace, now is the time to send him our website address. www.curetay-sachs.org. Our work is never too late for our kids and those kids yet to be born. They deserve a full and happy life. That's why we do this"
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.
The CTSF had over 400 different donors in January through March. All of these folks chipped in to help us to continue our research funding! We had 17 fundraisers!! 3 of those were local events, and 13 were done on Facebook. Thanks to the Andersons, Salonys and Luces for their efforts and to everyone who set up an easy FB Fundraiser to celebrate some special occasion for us. We had 8 Corporate Matches and got donations from 11 United Way agencies that we either state or locally based in the US. Donations came from all across the world and in memory and/or honor of 32 of our kids. And to our friend Peggy Moffit in Heaven, we got so many loving donations in your memory! You were very loved here on earth. Rest in peace our friend.
Although the numbers are not fully baked yet, we brought in just shy of $115,000 for the quarter. We also made some additions to our Board of Directors. Did you know that everyone on our Board, the folks that make our investment decisions, are people who are blood relatives of a Tay-Sachs or Sandhoff child? Simply stated, they have or are living it, and they get it!
When the CTSF started, there basically was zippo in terms of research. Today, I talk regularly with leading researchers about Gene Therapy, Clinical Trials on real kids, FDA approvals, UK/and the European Union funding, Tanganil, and "Compassionate Care" laws. Yep, it's complicated. But the bottom line is that several research efforts are all coming together at once. So much will happen in the next 8-10 months. When I get the "OK" from several of the researchers that we have funded, you will be the first to know the details. All of this has happened because of your help.
We are too late for too many kids, including mine. But, as Churchill spoke to Parliament when it looked like the Nazi's would successfully invade, he said... "We shall never surrender". With your help, we won't either.
- Rick, and the CTSF Board
Nikko was born on May 14 2015 and he is our firstborn son. Nikko since day one I always knew as his mother that he was angelic and filled a room with so much love with just his presence. What I didn't know was Nikko would be
Words cannot express what a privilege it was to be Brennan's parents. The Lord chose us to care for this precious child and now he is home. Brennan passed on March 4, 2016, in the comfort of his own home. It was fairly sudden and our hearts are broken. Brennan's parents, Holly and Royce Stringer, reside in Prescott, Arizona.
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.
My name is Alexis Nichole Markowich or just "Lexi". I was born on March 9, 2001 to Craig and Nichole Markowich and I also have a big sister Brianne. When I was four, on August 4, 2005, we got the devastating news that I had Juvenile Onset Tay Sachs Disease (JOTSD).
Isabel was born in Buenos Aires on February of 2009. Until the age of two Isabel was a healthy little girl. She had the life that any other child could have: school, little friends at park, holidays and trips to Spain to visit her family. And always a big smile on her face...
Our daughter Lexi was born on March 30, 2011. She was born in Junction City, Kansas right outside of Fort Riley. I am in the army so.....
Our son Nathan Harney is almost 17 months old. He is a wonderful, happy baby who has beautiful blue eyes, a huge personality, and is the light of our lives. He is a gentle soul that we have been blessed with......
My sisters and I had what seemed to be a normal up bringing in Southwestern, Ohio to a set of parents who were regular too.....
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.
Kaiden was born December 20, 2006. He was my beautiful blue eye, blond hair angel. He was the best baby any parents could ask for. He smiled constantly and barely cried. I would tell my Mother all the time that he was my "special little man".......
Elise was born December 1, 2004 in Santa Maria, California. She was an absolutely perfect baby with an easy pregnancy. We never imagined we could love someone as much as we love Elise. The next 6 months were the happiest months of our lives......
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.....
Dylan James Manning (commonly referred to as "DJ") was born on Thanksgiving Day, November 22, 2001. Dylans arrival was a long awaited event for his parents, Brian and Sherri of Atglen, Pennsylvania. Although Dylan was anxious to arrive almost four weeks early, he was not quick to reach his milestones. Doctors naturally gave him a little extra time to reach things like crawling and rolling over due to his prematurity.
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.
My name is Lacie Wivell and this is my story. I was born on April 8, 2004, to Rick and Lisa Wivell. I had two older sisters, Amanda and Rikki, and two older brothers, Levi and Colby. I also had another brother, Dakota, that went to be an angel in 2001.
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The Cure Tay-Sachs Foundation is very grateful you decided to donate and we thank you for your generosity. Your donation will be used to support research to find a cure for Tay-Sachs disease.
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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