ECHS1
Seeking a cure for echs1 deficiency
HELP US REACH OUR CURRENT $1.5M GOAL!
Your support is crucial in raising awareness and funds for gene therapy pre-clinical research, preparation for a Toxicology Study, or whatever exciting initiatives may be next.
The sooner we reach our goals, the faster we get to a potential Clinical Trial.
See our Fundraising Progress below.
Thank you for your SUPPORT
Gene therapy is a revolutionary approach to treating genetic diseases, where a one-time treatment can provide a lifetime of benefits. Instead of treating just the symptoms, gene therapy fixes the disease at the source by replacing the defective gene with a healthy copy.
A healthy copy of the patient's defective gene is loaded into a virus that has been stripped of its own DNA.
Trillions of viruses, each containing a healthy copy of the gene, are injected into the patient's spinal fluid.
The viruses bind to cells in the patient's spinal cord and brain and deliver healthy gene's to the cell's nucleus.
FUNDRAISING PROGRESS
ECHS1 FUNDRAISING VILLAGE
It truly takes a village.
Families from around the world are partnering with Cure Mito Foundation to raise funds that get us closer to a treatment or cure for our children.
If you are interested in supporting a fund which honors a child or family member affected by ECHS1 Deficiency, please click the "DONATE" button associated with one of the campaigns below.
Please email info@curemito.org if you would like information about starting a fund in honor or memory of your loved one.
Thank you for supporting our children!
