Episode 19

Published on:

6th Oct 2021

Sifting Through Too Many Options

We have been able to share some exciting news in the last two episodes. Sanath and cureGPX4.org commissioned a High-Throughput Screening assay to look for compounds that improve the growth of Raghav’s cells in culture. Then we heard the exciting news that the assay found 116 hits, 43 of which are already approved for other conditions.

Sanath has pulled together a team of experts to help him sift through this list of compounds and develop a plan for systematically assessing the various properties of these compounds. This is where the hard work begins. Approved medicines will have loads of data describing these properties. But there is much less data publicly available for investigational compounds.

Either way, the decision about what to try next is not going to be easy

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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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