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This is Harish Gaddam, raising funds on behalf of my brothers family - Suresh & Harini for their 3 year old little girl - Parvi Gaddam. She has been diagnosed with blindness of a rare genetic mutation : LCA3 (SPATA7), which has no FDA approved treatments or trials in progress.
After tons of sleepless nights reaching out to each and every renowned eye hospitals, doctors and various scientists almost all around the world we came in touch with Dr. Rui Chen, Baylor College of Medicine who had successfully done mouse model for LCA3. He has made us realize that LCA3 Gene therapy is a really effective treatment and showed promising results which can warranty the advancement to primates and clinical trials.
Long Term Goal
For taking LCA3 gene therapy to clinical trials, it needs collaboration with researchers, trial investigators , doctors, laboratories, pharmaceutical companies and others. It needs at least 5 million dollars to conduct a trial and to send for FDA approval.
As per the estimates we have received from scientists and researchers we can complete preclinical for around 500,000$ . Trying to reach this goal with this Milaap fund raise campaign. This is going to help my kid and many others around the world to see !.
We have reached out to several pharmaceutical companies, doctors, researchers and had discussions with multiple foundations . But their motto is simple ! They can't spend any effort/money on patients with rare gene as the impact is less.
The only option we have is to go for a crowd fund raising to help my daughter see this beautiful world. Researchers are ready to do this in pre-clinical if we get sufficient funding.
We are here with loads of hopes and prayers raising funds for advancing to trials. We appreciate and never forget the helping hands in our tough times.
Gene therapy treatment can literally make blind see stars !
Refer article , similar Gene therapy treatment for different gene made wonders and kids treated are able to see planes and stars for first time in their life.
Please donate by pouring your heart out ! We need generous contribution from each one of you, it will help us move a step ahead in the goal. Hoping Parvi and many other kids will see the world one day just like us with your kind contribution!
Please follow and share our story to your near and dear ones on social media.
UrSureshGaddam - Twitter Handle
