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halukakinon Ask HN: How to raise funds for rare disease research?

Thank you for sharing this. I think we should add such organizations' links to idefine website. In this case, I'm guessing not all Australian parents would know about this org.

halukakinon Ask HN: How to raise funds for rare disease research?

That's a good question. Several days/hours worth of PT. Hospital visits/stays every few months. Lab work. That should add up to something for the insurance companies.

halukakinon Ask HN: How to raise funds for rare disease research?

Received it. Thank you Trey.

halukakinon Ask HN: How to raise funds for rare disease research?

Definitely! I have a few creative agencies in mind to get in touch with.

halukakinon Ask HN: How to raise funds for rare disease research?

That's my layman question as well since the first covid injection. Maybe my daughter getting a shot everyday/week/month? Like insulin injections.

halukakinon Ask HN: How to raise funds for rare disease research?

Thank you! I don't see the companies as evil. I understand how profitable it is for Novartis to sell the SMA drug. But somehow they do not touch the whole thing until the drug is ready to use. I'll read the articles.

halukakinon Ask HN: How to raise funds for rare disease research?

"How to raise funds"

halukakinon Ask HN: How to raise funds for rare disease research?

First time I hear about them. Looks promising. We'll put this on our list. Thank you!

halukakinon Ask HN: How to raise funds for rare disease research?

That's a very good idea. I guess we could send out letters to them.

halukakinon Ask HN: How to raise funds for rare disease research?

Yes definitely. Indeed I'm writing this to understand how we can do this for idefine. I wrote it in my post above. Sorry, I understand the long post made it hard to follow.

halukakinon Ask HN: How to raise funds for rare disease research?

Yes, it turns out GLP is a very important protein. The second paper I cited above makes it sound it is pretty easy to supplement mice with GLP.

halukakinon Ask HN: How to raise funds for rare disease research?

That is actually very smart. Right now we are limited to Netherlands and US mostly. Let me read about Canada.

halukakinon Ask HN: How to raise funds for rare disease research?

Got it. Totally understand what you mean here. Will definitely try this.

halukakinon Ask HN: How to raise funds for rare disease research?

Will certainly do. Thank you Alok!

halukakinon Ask HN: How to raise funds for rare disease research?

Sometimes I share this feeling, but then I think about all the loved ones who were cured of some disease that would have been deadly 50 years ago. I don't think it's the pharma companies that are responsible for this. They are for-profit entities. Our governments should create the necessary business environment which will encourage these companies to work more for the people.

halukakinon Ask HN: How to raise funds for rare disease research?

Wow. So glad for you! These rare disease stories with a positive ending give us all the hope. Best of luck!

halukakinon Ask HN: How to raise funds for rare disease research?

Considering they managed to raise close to $100M between them, if we could even get 1-2% cross donations from their existing donors that could give us a big jump start. This is an area I think we will definitely look into.

halukakinon Ask HN: How to raise funds for rare disease research?

I believe they call it "derisking" for the companies. It's a topic mentioned a lot on Ethan's genefixers clubhouse meetings. As you summarized, it's a very important topic. For anyone interested, the episode with Dr Allyson Berent is a masterclass about this. https://www.clubhouse.com/room/Md8njknG?utm_medium=ch_room_x...

halukakinon Ask HN: How to raise funds for rare disease research?

I first heard about her efforts when she donated $40M to UCF. https://www.ucf.edu/news/scott-40-million-transformational-i...

I hope we can get in touch somehow. Thank you.

halukakinon Ask HN: How to raise funds for rare disease research?

Thank you Matt! Just so you know, "quickstart molecular biology" is my daily reading thanks to you.

halukakinon Ask HN: How to raise funds for rare disease research?

From a software developer perspective: A github like service where every incremental research step is recorded&visible. A build management system like travis where each experiment is built and held accountable to unit tests. Something like github actions where you can trigger an automated lab trial instantly. Somehow opensource SW development communities have so much they can teach to medical researchers in terms of how to scale development.

halukakinon Ask HN: How to raise funds for rare disease research?

I wouldn't know the first thing about medical drugs. But I noticed, interestingly, Ketamine is a mentioned drug in the first research paper I cited above.

halukakinon Ask HN: How to raise funds for rare disease research?

Glad to hear a good story. Sobering is a very good way to put it. For some of the parents, it just brings something else out of them. You lose the option to be a couch potato, forever.

halukakinon Ask HN: How to raise funds for rare disease research?

Thank you! Thanks to Ethan and Julia's weekly meetings on clubhouse, I've heard about some of these. Alok's DAO-based funding system seems very promising. I hope that gets traction. I thought I knew more about RareBase, but now I think I'll need to study their site in more depth.

halukakinon Ask HN: How to raise funds for rare disease research?

These are great leads. I will study them. Thank you.

halukakinon Ask HN: How to raise funds for rare disease research?

That's a big tip for the down the road. Will try to understand this better. Thank you.

halukakinon Ask HN: How to raise funds for rare disease research?

Thank you. Great job btw, lifebit could do some real good! Same idea for me too. If I can manage to make a clean exit on my existing business, medicine R&D will be where I will spend the rest of my time.

halukakinon Ask HN: How to raise funds for rare disease research?

Thank you so much. First time I hear about Kennedy Krieger. Their "feeding disorder" service caught my eye. That's a big issue for us. They post some pretty good results. https://www.kennedykrieger.org/outcome-data-2019/feeding-dis... . I'll study the CRND site as well.

halukakinon Ask HN: How to raise funds for rare disease research?

This looks very helpful. First time I heard about this organization. Apparently, they train patient groups like us. They even host their own drug repurposing conference. I'll signup on the email list and study their site. Thank you.

halukakinon Ask HN: How to raise funds for rare disease research?

We couldn't get in last year's batch. I'm thinking we will re-apply this year. If any CZI team members read this at some point. Any pointers for the future would be a great help.