Where the Work Gets Done...
News Stories about n-1 drug therapies
October 25, 2020
Let me begin by saying that finding a cure for an incredibly rare disease used be unthinkable.
But with today's technologies, these n-1 cases ( meaning research cases being done on only one individual) are becoming more and more available and bringing hope to those who suffer now, and in the future, from these rare debilitating diseases.
I've included links to two specific groups who are the biggest hope for us at The Foundation for Casey's Cure, because while we may be looking into one subgroup, that research often overlaps other researchers' work, thereby spreading complimenting therapies for other rare disorders.
One organization is the Charles River Lab, who was able to support @TimYu 's work at Boston Children's hospital and find a cure within 7 months of his initial search. That wasn't possible because he alone constructed the cure from scratch. Rather, he built upon those who are in current searches for rare diseases and was able to build this cure from that foundational information.
Another organization that is the n-Lormen Foundation :
n-Lorem Foundation launches to develop individualized therapies for ultra-rare diseases.
Led by former Ionis CEO Stanley Crooke, the nonprofit hopes to make it easier, safer, and cheaper to
develop N-of-1 drugs
This is recent work that we are hoping will help us raise the initial $50,000 (out of a total of $350,000) in order to get research started with The Charles River Lab, and Dr. Tim Yu's Lab with Dr Granzier from Ohio State.
You can help us by donating through our Charitable Website. And remember, every contribution counts!
https://charity.gofundme.com/o/en/campaign/finding-caseys-cure3
Let me begin by saying that finding a cure for an incredibly rare disease used be unthinkable.
But with today's technologies, these n-1 cases ( meaning research cases being done on only one individual) are becoming more and more available and bringing hope to those who suffer now, and in the future, from these rare debilitating diseases.
I've included links to two specific groups who are the biggest hope for us at The Foundation for Casey's Cure, because while we may be looking into one subgroup, that research often overlaps other researchers' work, thereby spreading complimenting therapies for other rare disorders.
One organization is the Charles River Lab, who was able to support @TimYu 's work at Boston Children's hospital and find a cure within 7 months of his initial search. That wasn't possible because he alone constructed the cure from scratch. Rather, he built upon those who are in current searches for rare diseases and was able to build this cure from that foundational information.
Another organization that is the n-Lormen Foundation :
n-Lorem Foundation launches to develop individualized therapies for ultra-rare diseases.
Led by former Ionis CEO Stanley Crooke, the nonprofit hopes to make it easier, safer, and cheaper to
develop N-of-1 drugs
This is recent work that we are hoping will help us raise the initial $50,000 (out of a total of $350,000) in order to get research started with The Charles River Lab, and Dr. Tim Yu's Lab with Dr Granzier from Ohio State.
You can help us by donating through our Charitable Website. And remember, every contribution counts!
https://charity.gofundme.com/o/en/campaign/finding-caseys-cure3
January, 2020
This page is intended to give our prospective donors an understanding of who we believe will be the ultimate researchers on Case's defective gene, #603689, located on the TTN gene, the longest protein chain in our bodies.
These links, while not comprehensive, is intended to provide some insight into the work being done in the area of neurological diseases, where the study of Muscular Dystrophy resides.
This page is intended to give our prospective donors an understanding of who we believe will be the ultimate researchers on Case's defective gene, #603689, located on the TTN gene, the longest protein chain in our bodies.
These links, while not comprehensive, is intended to provide some insight into the work being done in the area of neurological diseases, where the study of Muscular Dystrophy resides.
- https://wayback.archive-it.org/7993/20190207210420/https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm
- Dr. Kim Yu's Work on Duchenne Muscular Dystrophy: kim_yu_nejm-2019_patient-customized_aso.pdf
- NORD: Information from the National Organization for Rare Diseases Research Policy: nord_rare_disease_research_grant_policy-1.pdf
Part of an email received from our first outward request for researchers interested in studying Casey's tissues:
Our board member (a graduate of Notre Dame University) , heard from Ohio State (researchers) today (March 4, 2020) and they have an approved protocol to use out-of-state patients. Recall this was the collaboration with Dr. Granzier to study TTN protein expression and function in our cohort of TTN patients at Ohio State. The ideal match would be Casey and Christopher, (Casey's unaffected brother). .In addition to the biopsy, blood specimens can be taken for work on specific cells. I could see the latter being done at Granzier’s lab in Arizona.
Now Granzier has decades of work on TTN with a functional lab. Yu mentioned he would be happy to talk with him about his work on oligonucleotides which could be a fruitful collaboration to get some direct (applied?) research on Casey’s cells. Our Board Member will try to blind contact the CEO and chief R&D folks at Sarepta (a medical company currently working on Duchenne and Limb-Girdle muscular dystrophies) with our issue (useful as a lab with muscle application). To me (the Board Member), this can form a rudimentary team. I am thinking if you can raise the money we might get Granzier started on some targeted work.
Our board member (a graduate of Notre Dame University) , heard from Ohio State (researchers) today (March 4, 2020) and they have an approved protocol to use out-of-state patients. Recall this was the collaboration with Dr. Granzier to study TTN protein expression and function in our cohort of TTN patients at Ohio State. The ideal match would be Casey and Christopher, (Casey's unaffected brother). .In addition to the biopsy, blood specimens can be taken for work on specific cells. I could see the latter being done at Granzier’s lab in Arizona.
Now Granzier has decades of work on TTN with a functional lab. Yu mentioned he would be happy to talk with him about his work on oligonucleotides which could be a fruitful collaboration to get some direct (applied?) research on Casey’s cells. Our Board Member will try to blind contact the CEO and chief R&D folks at Sarepta (a medical company currently working on Duchenne and Limb-Girdle muscular dystrophies) with our issue (useful as a lab with muscle application). To me (the Board Member), this can form a rudimentary team. I am thinking if you can raise the money we might get Granzier started on some targeted work.