RESEARCH
Today, there are limited treatments for muscular dystrophy, leaving parents and children with few options. Current treatments, such as steroids and physical therapy, only help in limited ways.
Ultimately, MD robs patients and families of life itself. Even with newly approved drugs like Endosys 51 (this is a gene therapeutic that specifically targets those individuals with very specific mutations) the increases in dystrophin are limited. While any improvement is monumental, we believe we can do more!
Enter CRISPR, the promise of which cannot be overstated. CRISPR provides the ability to fix mutated genes, like dystrophin, allowing the body to produce it and live a normal life. Strongest Hearts is funding research that could treat nearly 90% of people affected by Duchenne’s or Becker’s.
Ultimately, MD robs patients and families of life itself. Even with newly approved drugs like Endosys 51 (this is a gene therapeutic that specifically targets those individuals with very specific mutations) the increases in dystrophin are limited. While any improvement is monumental, we believe we can do more!
Enter CRISPR, the promise of which cannot be overstated. CRISPR provides the ability to fix mutated genes, like dystrophin, allowing the body to produce it and live a normal life. Strongest Hearts is funding research that could treat nearly 90% of people affected by Duchenne’s or Becker’s.
PROGRAMS
Phase 1: Funding Research
Strongest Hearts first grant is being given to Hunterian. Hunterian is an industry leading CRISPR lab, focused on using CRISPR to create therapies for the worst genetic diseases.
Founded by Dr. Vinod Jasuka-Ranga, Hunterian is dedicated to the eradication of DMD and BMD and the use of CRISPR to attack other types of muscular dystrophy in the future.
Strongest Hearts chose Hunterian to work with, based on the founders’ desire to not only focus on a therapy to help only their son’s situation, but to help as many if not all children with these dystrophies.
The grant will go to the pre-clinical trials and will validate the use of Hunterian’s methodology and treatment. Future phases will go towards the ultimate goal of clinical trials and a FDA approved therapy.
Founded by Dr. Vinod Jasuka-Ranga, Hunterian is dedicated to the eradication of DMD and BMD and the use of CRISPR to attack other types of muscular dystrophy in the future.
Strongest Hearts chose Hunterian to work with, based on the founders’ desire to not only focus on a therapy to help only their son’s situation, but to help as many if not all children with these dystrophies.
The grant will go to the pre-clinical trials and will validate the use of Hunterian’s methodology and treatment. Future phases will go towards the ultimate goal of clinical trials and a FDA approved therapy.
Phase 2: Advocacy and Testing Program
Strongest Hearts is designed with a mission to cure DMD and BMD. While we work towards that goal, we realize there are many families who have not been tested or have access to testing.
Strongest Hearts is working to create a program that will help offset the cost of genetic testing, and blood tests designed to help identify and diagnose the disease. As a genetic disease, DMD and BMD are nearly always hereditary (there are very limited cases of first time mutations) and as a result once one individual is diagnosed, there is a very high likelihood there are other family members either afflicted by, or carriers of the disease.
Testing is crucial for these individuals and families, not only to begin dealing with the disease, but to seek proper genetic counseling and to help prevent the future generations from suffering with the disease as well.
We believe that by attacking DMD and BMD along all of these fronts, we can make a dramatic difference in the way the disease is treated, identified and passed.
Strongest Hearts is working to create a program that will help offset the cost of genetic testing, and blood tests designed to help identify and diagnose the disease. As a genetic disease, DMD and BMD are nearly always hereditary (there are very limited cases of first time mutations) and as a result once one individual is diagnosed, there is a very high likelihood there are other family members either afflicted by, or carriers of the disease.
Testing is crucial for these individuals and families, not only to begin dealing with the disease, but to seek proper genetic counseling and to help prevent the future generations from suffering with the disease as well.
We believe that by attacking DMD and BMD along all of these fronts, we can make a dramatic difference in the way the disease is treated, identified and passed.
Phase 3: Education
Education Program- Strongest Hearts is dedicated to education. As outlined in the about us section, the reality is that many families spend years looking for answers.
Even when families do find a diagnosis, they are left to do much of the research on their own, with no idea on how to begin even tackling something as daunting as treatment for a condition like DMD or BMD.
Strongest Hearts is dedicated to helping families, physicians and physical therapists understand the disease, what the realities are and how to find out about it much earlier. Early detection can help families deal with the large financial planning and burden, makes plan for care, avoid potentially harmful activities and give peace of mind and guidance.
Even when families do find a diagnosis, they are left to do much of the research on their own, with no idea on how to begin even tackling something as daunting as treatment for a condition like DMD or BMD.
Strongest Hearts is dedicated to helping families, physicians and physical therapists understand the disease, what the realities are and how to find out about it much earlier. Early detection can help families deal with the large financial planning and burden, makes plan for care, avoid potentially harmful activities and give peace of mind and guidance.
