Support Raghav's Cure - Rare disorder in GPX4 gene
Donation protected
Indian friends can donate using this link - https://milaap.org/fundraisers/support-raghav-5
Raghav has a rare change in DNA — a change in GPX4 gene that has a 1 in 75 million chance of occurring.
He is 1 year old but cannot sit up without support. He doesn’t have the muscle strength to hold a toy in his hand. He doesn’t have the oral strength to swallow food. He cannot hear normally. His bones are not developing as expected. We don’t really know what his future looks like.
No Treatment Today
As of today, there is no treatment for Raghav. Our only option is to wait until his symptoms get severe, give him some pain killers, and comfort him.
But there is hope - Research
We are partnering with Dr. Russell Saneto , a Professor of Neurology at University of Washington and an expert on Mitochondrial Diseases. His team is going to recreate Raghav’s mutation on a mouse and examine the effects. This is the first step towards understanding more about this disease, identifying potential drug candidates, partner with pharmaceutical companies to test them on mouse, submit a proposal to FDA, conduct clinical trials and eventually administer the drug to Raghav.
Here is a quick run down of the steps involved in finding a cure:
Phase 1: Create a mouse model (oh, btw find researchers before that)
Phase 2: Conduct research studies on the mouse with candidate drugs
Phase 3: Go through FDA process for approval
Phase 4: Check if the medicine works for Raghav (..and we could end up in Phase 2 all over again at this point if it fails)
This could take several years, millions of dollars, and hundreds of people working on it. I don’t know if I will succeed. But I want to take the first step and cross the remaining bridges when I get to it.
Donate Now
Time is of essence. The earlier we treat Raghav the lesser damage to his body.
After growing up, Raghav might become a doctor, scientist, singer, actor, or a Nobel laureate. But whatever he does, I will teach him to be kind and give back to the community that made his life a reality. You are a part of making this dream a reality. Each dollar takes Raghav one step closer to living life the way you and I do.
Be generous.
Be kind.
Be the miracle Raghav needs.
THANK YOU ❤️
Raghav has a rare change in DNA — a change in GPX4 gene that has a 1 in 75 million chance of occurring.
He is 1 year old but cannot sit up without support. He doesn’t have the muscle strength to hold a toy in his hand. He doesn’t have the oral strength to swallow food. He cannot hear normally. His bones are not developing as expected. We don’t really know what his future looks like.
No Treatment Today
As of today, there is no treatment for Raghav. Our only option is to wait until his symptoms get severe, give him some pain killers, and comfort him.
But there is hope - Research
We are partnering with Dr. Russell Saneto , a Professor of Neurology at University of Washington and an expert on Mitochondrial Diseases. His team is going to recreate Raghav’s mutation on a mouse and examine the effects. This is the first step towards understanding more about this disease, identifying potential drug candidates, partner with pharmaceutical companies to test them on mouse, submit a proposal to FDA, conduct clinical trials and eventually administer the drug to Raghav.
Here is a quick run down of the steps involved in finding a cure:
Phase 1: Create a mouse model (oh, btw find researchers before that)
Phase 2: Conduct research studies on the mouse with candidate drugs
Phase 3: Go through FDA process for approval
Phase 4: Check if the medicine works for Raghav (..and we could end up in Phase 2 all over again at this point if it fails)
This could take several years, millions of dollars, and hundreds of people working on it. I don’t know if I will succeed. But I want to take the first step and cross the remaining bridges when I get to it.
Donate Now
Time is of essence. The earlier we treat Raghav the lesser damage to his body.
After growing up, Raghav might become a doctor, scientist, singer, actor, or a Nobel laureate. But whatever he does, I will teach him to be kind and give back to the community that made his life a reality. You are a part of making this dream a reality. Each dollar takes Raghav one step closer to living life the way you and I do.
Be generous.
Be kind.
Be the miracle Raghav needs.
THANK YOU ❤️
Organizer and beneficiary
Ramya Ramaswamy
Organizer
Bellevue, WA
Sanath Kumar Ramesh
Beneficiary