Olga was born on November 3, 2017. First 3 months, she was growing up as a healthy newborn baby. But at the age of 4 months, she had extremely poor head control. During the next 3 months, Olga was checked by many different doctors and had many passible analyzes, tests, and medical examinations, and at the age of 7-month-old, she was diagnosed with Canavan disease.
The Canavan Disease is a very rare fatal genetic neurological disorder characterized by the spongy degeneration of the white matter in the brain. Affected infants may appear normal at birth, but usually develop symptoms between 3-6 months of age. Canavan disease belongs to a group of disorders known as the leukodystrophies. The leukodystrophies are a group of rare, progressive, metabolic, genetic disorders that can affect the brain, spinal cord and often the nerves outside the central nervous system (peripheral nerves)
The prognosis is extremely poor - most affected children develop life-threatening complications by 10 years of age. Being 22-month-old, Olga can´t crawl, sit, walk, she does not know how to play with toys, she cannot even hold her head up. But she is a very smiling little angel! Olga really wants to learn all the habits, but she cannot ...
A group of scientists at Rowan University School of Osteopathic Medicine, New Jersey led by Paola Leone, Ph.D. (Neuroscience), developed a method of gene therapy to treat the Canavan Disease. This is a completely new technique already successfully used to treat some other genetic diseases. In gene therapy, healthy copies of the defective gene are inserted by deactivated adenovirus into the brains of affected children. Children treated with gene therapy have shown marked improvement of symptoms. But, the positivity of the result of gene therapy completely correlated with the age of a child, the earlier the therapy starts the better treatment effect can be obtained..
Unfortunately, because Canavan disease is very rare, the pharmaceutical companies refuse to finance the development and clinical trials of treatment, and it is possible to conduct them only at the expense of private funds. Olga was taken into the program, but the price is very high - $1,140,000.
Please, help us raise the funds for the treatment of Olga!
Any reasonable amount, $10, $20, $50 will help us get closer to our goal. Your contribution will make an important impact. It will help us SAVE little Olga. If you participate in our fundraising efforts you will become a part of something truly inspiring.
We are inviting you to be a part of this future
and help us SAVE Olga and other kids with Canavan disease.
WHERE YOUR MONEY GOES:
All money raised in this fundraiser will go to 501(c)(3) nonprofit organization, Canavan Research Foundation (CRF). The mission of the Canavan Research Foundation is to fund and support research to cure Canavan disease and apply these findings to create therapies for other genetic brain diseases. All charitable donations are provided free of charge and will help to treat Olga and other kids with Canavan disease.
- Chiara Gentiloni
- Anastasia Semenova
- Irina Krasnyanskaya