Podknife - Hope for Ultra-Rare Diseases by Soonish

Help
Help

Suggested Topics
- What is Podknife?
  
  Podknife is a curated podcast information and review site designed to be accessible in your browser from any device. We’re working to build the most useful podcast information source available by providing you with as much publicly available information about each podcast in our database as we can find and keeping it as up to date as possible.
- Can I listen to podcasts on Podknife?
  
  Yeah! There's a player at the bottom of the page.
- How can I get my podcast on Podknife?
  
  We’d love to hear about your podcast and consider it for addition. You can suggest your own by logging in and selecting the “Suggest a Podcast” link from the menu. Fill out the form and we’ll take it from there.
- How can I submit a review of a podcast?
  
  To submit a review of a podcast, go to the page of the podcast you want to review and click the "Submit Review" button below the image associated with the podcast. Clicking that button brings you to a new page where you can select a rating between 1 and 5 stars and write a brief review.
- How can I submit a review of a podcast episode?
  
  To submit a review of a podcast episode, go to the page of the podcast whose episode you want to review and find the specific episode you're looking for. Clicking on any episode reveals a "View Episode" button and clicking on that brings you to an episode-specific page where you can find more information displayed about the episode along with a "Submit Review" button. Clicking that brings you to a new page where you can select a rating between 1 and 5 stars and write a brief review of the episode.
- Is there a Podknife app?
  
  Not yet - we're keeping it in the browser at least for now. No app download required to access anything on the site.
- How can I change my display name?
  
  To change your display name, log in to Podknife and select "Account Settings" in the dropdown menu. From the Account Settings page, enter your desired new display name and press the "Save" button to confirm the change.
- How can I change my password?
  
  To change your password, log in to Podknife and select "Account Settings" in the dropdown menu. From the Account Settings page, enter your desired new password in the two fields below "Update Password" and press the "Save" button to confirm the change.
- How can I keep track of all my reviews?
  
  Within the menu, click on "My Profile", you will then be able to check all your reviews for both podcast & episode. You can also check all your favorite podcasts.
- How can I keep track of all my favorited podcasts?
  
  You can find podcasts you’ve favorited displayed on the My Profile page associated with your account (https://podknife.com/profile).
  You can share your My Profile page displaying your favorite podcasts as well as any reviews you’ve written publicly with a link in the form https://podknife.com/users/[displayname] where [displayname] is replaced by your Display Name.
- How can I favorite a podcast?
  
  On each podcast page you’ll find the outline of a star above the podcast title. Clicking this star fills it in and designates the podcast as a favorite (for logged in users) or invites the user to log in or register for an account (for users who are not logged in). You can find favorited podcasts displayed on the My Profile page associated with your account (https://podknife.com/profile).
- How can I register for a Podknife account?
  
  To register, click on the “Register” button in the top right of your browser window (or visit https://podknife.com/users/sign_up). Enter a Display Name to be associated with your account on the site (alongside any reviews you contribute, for example), an Email address to associate with the account and your password (twice for confirmation). Press the “Confirm” button and you should be all set, logged in and ready to review and favorite podcasts as you wish.
- How can I get in touch?
  
  We’d love to hear from you. You can reach us using the Feedback form (https://podknife.com/feedbacks/new) which can be found either in the footer on each page (for users who are not logged in) or in the dropdown menu (for logged in users).
  
  You can also reach us on Twitter by tweeting us @podknife (https://twitter.com/podknife) or on Facebook by messaging us from our page (https://www.facebook.com/podknife/).
- Log In
- Register
- Feedback
- Help
- Privacy Policy
- Terms of Use
© Podknife 2024

This episode currently has no reviews.

Hope for Ultra-Rare Diseases

Podcast |: Soonish
Publisher |: Hub & Spoke
Media Type |: audio
Podknife tags |: Society & Culture,; Technology
Categories Via RSS |: Documentary,; Science,; Society & Culture,; Technology
Publication Date |: Apr 30, 2021
Episode Duration |: 00:45:20

Description
iTunes Summary

In this episode of Soonish you'll meet Stanley Crooke, the former CEO of Ionis Pharmaceuticals and the head of a new nonprofit called N-Lorem, which is working to make mutation-correcting "antisense oligonucleotide" drugs available free for life to people with uncommon genetic diseases.

These are conditions so rare they often don't have a name. But while the diseases themselves are unusual, the problem isn't: as many as 350 million people worldwide are thought to carry mutations that give rise to unique "N of 1" health problems.

The debut of hyper-personalized antisense medicines is a topic I covered in a March 2020 episode of the podcast Deep Tech for MIT Technology Review. Back then, N-Lorem was just getting started. So I was excited to connect with Crooke one year later and go into more depth how antisense drugs work, why they're well-suited for treating some genetic diseases, and how Crooke realized he could give some patients personalized versions of these drugs for free—and for life.

"It was literally impossible until just now," Crooke says. Listen to find out what changed—and what it could mean for the future of drug discovery and the way we regulate and pay for advanced therapies.

For more, head to soonishpodcast.org, where we've got the full transcript and additional resources.

Notes

The Soonish opening theme is by Graham Gordon Ramsay. All additional music by Titlecard Music and Sound.

If you enjoy Soonish, please rate and review the show on Apple Podcasts. Every additional rating makes it easier for other listeners to find the show.

Listener support is the rocket fuel that keeps our little ship going! You can pitch in with a per-episode donation at patreon.com/soonish.

We’ve spent decades trying to understand human biology, health, and illness at the level of our genes. For people with extremely rare genetic conditions, that work is finally starting to pay off. Thanks to the emerging field of hyper-personalized medicine, and the work of new organizations like the N-Lorem Foundation, we're entering a future where diseases linked to rare mutations don’t always have to be lethal.