#betathalassemia — Public Fediverse posts

#BBCNews - First gene-editing therapy may cure blood disorder
https://www.bbc.com/news/articles/c4gzldll44lo
#BetaThalassemia

NEW: #Vertex Pharmaceuticals is suing the gov over blocking access to #fertility preservation services for people getting #Casgevy, the first #CRISPR treatment, which treats #sicklecell & #betathalassemia

Currently, Vertex offering to help patients access these services illegal under anti-kickback laws

via @STAT
https://www.statnews.com/2024/07/15/vertex-casgevy-sickle-cell-infertility-hhs-lawsuit-fertility-preservation/

#health #healthcare #genetherapy #medicine

R to @EMA_News: The 1st gene editing therapy to treat #SickleCellDisease & #BetaThalassemia recommended for approval by EMA's human medicines committee, is also the 1st medicine using a novel gene-editing technology (#CRISPR/Cas9) in the 🇪🇺.

➡️ https://www.ema.europa.eu/en/news/first-gene-editing-therapy-treat-beta-thalassemia-and-severe-sickle-cell-disease

#CHMP #PublicHealth

🐦🔗: https://nitter.cz/EMA_News/status/1735660082413592605#m

[2023-12-15 13:56 UTC]

R to @EMA_News: The 1st gene editing therapy to treat #SickleCellDisease & #BetaThalassemia recommended for approval by EMA's human medicines committee, is also the 1st medicine using a novel gene-editing technology (#CRISPR/Cas9) in the 🇪🇺.

➡️ https://www.ema.europa.eu/en/news/first-gene-editing-therapy-treat-beta-thalassemia-and-severe-sickle-cell-disease

#CHMP #PublicHealth

🐦🔗: https://nitter.cz/EMA_News/status/1735660082413592605#m

[2023-12-15 13:56 UTC]

I would be remiss if I didn't plug Shelby's #YouTube channel here for a multimedia view of her experience with the gene therapy.

To quote Shelby, "Can you like and subscribe, give us a huge thumbs up, and smash that subscribe button?" https://www.youtube.com/watch?v=mdAck_FzsQs

#health #healthcare #medicine #scd #crispr #betathalassemia #transplant #genetherapy

But this is a slow-moving miracle. Shelby's #transplant itself took less than 8 minutes, but required more than 7 weeks in the hospital.

All told, from Shelby's #stemcell collection to the time she'll be allowed to leave immunity-protecting isolation and go back to in-person school, it will be over a year.

Read more: https://www.statnews.com/2023/12/13/gene-therapy-zynteglo-bluebird-beta-thalassemia/

#health #healthcare #medicine #scd #crispr #betathalassemia #chemo #patient

These #gene therapies are billed as "one time" therapies, basically miracle cures:

Almost 90% of the Zynteglo trial patients were transfusion-independent afterward, for at least 9 years.

The new #sicklecell treatments are similar. https://www.statnews.com/2023/12/08/fda-approves-casgevy-crispr-based-medicine-for-treatment-of-sickle-cell-disease/

#health #healthcare #medicine #scd #crispr #betathalassemia #transplant

I thought that the fantastical price tag might be keeping some patients from getting #Zynteglo

Turns out, that doesn't seem to be the case (yet), as I detail in a sister piece about Bluebird and the #insurance logistics behind these therapies:

https://www.statnews.com/2023/12/08/lyfgenia-sickle-cell-gene-therapy-bluebird-vertex-crispr-casgevy/

#health #healthcare #scd #crispr #betathalassemia #transplant #lyfgenia #casgevy #healthinsurance #pharma #bigpharma #biotech

I've been working on this story in some fashion since my first week at @STAT last Aug. The approval of Bluebird Bio's #genetherapy #Zynteglo for beta thalassemia caught my attention because

1) I myself carry the beta thal gene
2) The treatment is $2.8 million, at that time the most expensive drug ever

#drugprices #health #healthcare #scd #crispr #betathalassemia #transplant

It's been a long time coming...

Today is both Taylor Swift's birthday & the day I get to share the story of Shelby, a 7 (now 8) year old who's in the midst of a year+ "one-time" #genetherapy that will likely cure her #betathalassemia.

Don't miss the Taylor & Emily "The Space Gal" Calandrelli mentions, Shelby's budding #YouTube career, or how Sour Patch Kids are important for gene and #celltherapy:

https://www.statnews.com/2023/12/13/gene-therapy-zynteglo-bluebird-beta-thalassemia/

#health #medicine #patient #crispr #chemo #sicklecell #scd

A world first, a marker of progress
Gene therapy now approved in the UK
No more suffering
For sickle cell bearing
A medical breakthrough, of such success

#genetherapy #uk #sicklecell #betathalassemia #ode #poetry

https://www.medicalnewstoday.com/articles/uk-approves-gene-therapy-for-sickle-cell-disease-in-world-first