2/Over the past few months, we have done everything a parent would do. We read 100s of academic papers, built partnerships with scientists, translated foreign medical records, and self-compounded drugs at home...
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3/We dived into a technology called Antisense Oligonucleotide (ASO) that is *very* successful at silencing mutations at the source. Lydia’s treatment can be created within months! But this isn’t available to most children. Here’s why…
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4/There are 6 billion characters in our DNA. Everyone has typos in this code. Some typos cause serious diseases, like Lydia’s. There are millions with serious typos just slightly different from each other. To fix them, we need a customized drug for each patient.
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5/Traditional pharma isn’t set up to make $ from individual treatments. They have labeled these as “rare” and moved on. These are not rare. There are millions suffering. The tech exists; ASOs can be customized *within months*.
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6/We are on a mission. We have set up Lydian Accelerator to fill this gap. By open sourcing processes, tools and data, we can accelerate individual ASO treatments for all. With each treatment, we gather more data to reduce the time & cost.
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7/We are fortunate to be part of an incredible tech community. Help us save our little girl and pave the path for others devastated by genetic typos. Please DM me to chat about how you can help, retweet this, or donate directly on http://savelydia.com .
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8/I wrote a larger medium post with our story:https://medium.com/lydian-accelerator/saving-lydia-62a1c0bdf0fb …
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Thank you for all the love and support today. I should add, we are early in our journey. If anyone wants to brainstorm about best ways for a non-profit to open source knowledge and data, please DM me and I'd love to chat!
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Kraj razgovora
Novi razgovor -
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Have you done the whole genome sequencing of the all 3 of you ?
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We have exome sequencing data. Still waiting for full genome results.
- Još 3 druga odgovora
Novi razgovor -
Čini se da učitavanje traje već neko vrijeme.
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