Episode 7

full
Published on:

17th Apr 2021

Introducing Open Treatments: Making Rare Disease Research More Accessible

Sanath Kumar Ramesh – Rare Dad, Founder & CEO of Open Treatments, and Podcaster

The quest for treatments for rare diseases is challenging under the best circumstances. For ultra-rare diseases like Raghav’s, the system is just not built to find and provide treatments for nine patients. There is no viable business model. There is no regulatory pathway for approval when it is impossible to run clinical studies that can show statistical significance. If there is no approval, then insurance companies and even governmental payers like Medicaid are not going to pay the bill. Even if one could find a way to pay, how do you produce the medicines that emerge in such small amounts. 

This is the world that Sanath has been wrestling with since Raghav was born. Now he is doing something about it. He and his team have launched Open Treatments, an organization with a platform to make research and the R&D process accessible to families fighting these ultra-rare conditions.  The idea is to decentralize the research enterprise to individual diseases by pooling and sharing the tools, skills, processes, and knowledge about finding promising treatments. The vision is to find the treatments they need now and enable the hand-off of these treatments to commercial, governmental, and philanthropic entities that can ensure patients around the world have access to the therapies for years to come. 

In this episode, Sanath explains the origins and future of Open Treatments. (www.opentreatments.org )

Please support Raising Rare and the search for a treatment for Raghav by donating to Cure GPX4 


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About the Podcast

Raising Rare
Imagine the excitement of becoming a new parent and then within hours finding out your child has a serious developmental disorder.

That is exactly where Sanath Kumar Ramesh found himself in the summer of 2018. One year later, on his son’s first birthday, they found out that their son, Raghav, had an extremely rare mutation of the GPX4 gene. At the time, doctors told them that Raghav may be the only one on the planet with this genetic variant who had lived beyond one month of life. The prognosis is completely unknown.

They were alone.

They were scared.

And then they went into action.

And now they want to share their story.

Every couple of weeks Raising Rare will give you an update on baby Raghav as he grows up. We will also share how Sanath and his wife Ramya are driving toward a treatment for their son. We will explore the science that Sanath is initiating, their efforts to fund that research, and the people they meet along the way. We will also hear how the family adjusts to challenges and changes that they are faced with. Most importantly, we will share the wisdom they gain along the way so that other Rare Parents can learn from their steps and missteps.

We don’t know where this story will go. We do know we want you to join us for the journey.
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About your hosts

Sanath Kumar Ramesh

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Kevin Freiert

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