Dr Priya Sammani ( MBBS ,DFM )I often see worry etched on a parent's face when their little one isn't quite themselves. #anemia #BetaThalassemia #bloodtransfusion #geneticdisorder #HBBgene #hemoglobin #ironchelation
Kriszta Satori#BBCNews - First gene-editing therapy may cure blood disorder
https://www.bbc.com/news/articles/c4gzldll44lo
#BetaThalassemia
https://www.bbc.com/news/articles/c4gzldll44lo
#BetaThalassemia
Brittany TrangNEW: #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/
Liz HilesCOMPENSATED PATIENT EXPERIENCE OPPORTUNITY
Research Opportunity for caregivers of children between 12-25 diagnosed with Beta Thalassemia. 60-minute web-assisted phone interview compensation is $120
#BetaThalassemia #CooleysAnemia
https://rarepatientvoice.com/mirletaliz
Research Opportunity for caregivers of children between 12-25 diagnosed with Beta Thalassemia. 60-minute web-assisted phone interview compensation is $120
#BetaThalassemia #CooleysAnemia
https://rarepatientvoice.com/mirletaliz
EU Medicines AgencyR 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://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
New Accessory!
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:
#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