Cure Raghav - Rare disorder in GPX4 gene | Milaap
Cure Raghav - Rare disorder in GPX4 gene
62%
Raised
Rs.21,75,681
of Rs.35,00,000
1029 supporters
  • Sanath

    Created by

    Sanath Kumar Ramesh
  • R

    This fundraiser will benefit

    Raghav

    from Chennai, Tamil Nadu

Raghav is my sweet 2 year old son.

My biggest dream for Raghav is what most people consider to be mundane - for him to go to school, play in the park and eat ice cream like most kids do. Raghav cannot do any of this if we don’t find a treatment for his underlying genetic disease.

Raghav has an ultra rare genetic disorder called Sedaghatian type Spondylometaphyseal Dysplasia (SSMD). He is 2 years 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. His family really doesn’t know what his future looks like.

Raghav’s quality of life is not what one would want for any 2 year old. He went through 4 unplanned surgeries just in the last year. He goes through therapy to work on even basic skills such as swallowing his saliva. His future is described as wheelchair bound, non-verbal and prone to premature death.
Despite all this Raghav manages to give a smile, be it waking up from his crib or from an ICU bed. He is that resilient! Learn more about Raghav’s journey @ curegpx4.org

No treatment today, but there is hope!

As of today, there is no treatment for Raghav’s condition. Finding a new drug can take years, and we don’t think Raghav has the time for it. We have partnered with leading researchers to come up with practical short and long term strategies to find a treatment for his genetic disorder.

Short term: Drug Repurposing

We are working with researchers to comb through hundreds of approved medicines to test if any of those can be repurposed to treat Raghav’s condition. This research study costs $200,000 and is a very promising path to bring improvements to Raghav’s quality of life. Testing each drug costs $1500. By donating that amount you will open up the possibility of getting one drug to improve Raghav’s quality of life.

Long term: Gene therapy
Gene therapy will replace Raghav’s faulty gene with a good one. This is expected to fix the problem at the root cause. It costs $5-7 million and can take 4 years to get FDA approval. We are currently in the stage of developing a proof of concept for the gene therapy drug and testing its effectiveness on disease models.

Donate Now
Time is of essence. Raghav does not have a lot of time to wait and hope for a miracle to come along.

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 ❤️


Contact
Sanath (Raghav's Dad) - sanath@gpx4.org
Ramya (Raghav's Mom) - ramya@gpx4.org
https://www.curegpx4.org/

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