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Unveiling High-Throughput Screening Results
Episode 1818th September 2021 • Raising Rare • Raising Rare
00:00:00 00:29:50

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Since the moment they got the diagnosis of Sedeghatian-type spondylometaphyseal dysplasia (SSMD), Sanath and Ramya have been on a relentless quest to find something to help their son Raghav. They have tried some off-the-counter supplements. They worked with a small biopharma to write an IND to get access to an experimental compound. And recently, their non-profit CureGPX4.org commissioned a high-throughput study (HTS) of roughly 4000 compounds.

In this episode, we hear the results from that study.

The whole theory behind HTS is 'shots on goal'. The more shots you take, the more likely one of them will score. In the biopharma industry, HTS is done at an industrial scale with literally millions of shots on goal. Because SSMD is so rare, they have no idea what to expect from this relatively small sample. This is groundbreaking work.

And no matter what the results show, the work continues. Decisions will need to be made about what to try next. This is the episode you have been waiting for.