His sickle cell disease brought agony. Gene therapy is bringing hope. https://wapo.st/3NwqU1I This is such a wonderful read.
I was cured of #Hemophilia B with a gene treatment in Feb 2020. Life after pain is amazing. So glad #sicklecell cure is coming. Gene therapy is about to change medicine. #medicine

My latest Clinical Pipeline column for #NatureMedicine, on fitusiran, a small interfering RNA for the treatment of hemophilia A or B. It may be particularly useful for patients with inhibitors (antibodies against clotting factors).

#drugdevelopment #drugdiscovery #SciComm #sciencecommunication #pharma #hemophilia 👇

https://www.nature.com/articles/d41591-023-00037-3

Inequalities across 🇪🇺 remain one of the most important challenges for patients today.

Check out the @Parlimag article, which addresses further challenges and opportunities for #hemophilia patients 👇

https://www.theparliamentmagazine.eu/news/article/paving-way-for-another-decade-of-progress-in-hemophilia-care-through-eu-policy

🐦🔗: https://n.respublicae.eu/DeirdreCluneMEP/status/1649363476744220678

Bleeding disorders are not the end of the world; you could still lead a normal life. Stay healthy and be safe on this World Hemophilia Day!🩸

#GramyaAyurveda #WorldHemophiliaDay #WorldHemophiliaDay2023 #HemophiliaDay #Hemophilia

#OnThisDay First Earth-Size #Planet (Kepler-186f) Found In The 'Habitable Zone' of Another Star (2014).

Birth Anniversary of J. P. Morgan (1837) - American financier and #Banker.

Today is World #Hemophilia Day.

https://knowledgezone.co.in/news

AbbVie CEO Gonzalez scored $26M in 2022 pay as cash cow Humira neared patent cliff— What ancient art form has inspired many technological advances for medical devices? — FDA delays review of BioMarin's hemophilia gene therapy after data update-- See more on our front page news http://bit.ly/w28kSd #abbvie #pharmaceos #patents #devices #meddevice #medtech #productdesign #hemophilia #biomarin #genetherapy #pharma #pharmanews #biotech #biopharma #biotechnology #cafepharma

‘What’s the Value of a Life?’
At $3.5 million, Hemgenix, the new #genetherapy for #hemophilia B, has recently been named the most expensive drug on the planet, unseating another gene therapy, Skysona.
#Medicine #ethics
https://www.webmd.com/a-to-z-guides/news/20230215/life-saving-gene-therapies-cost-millions

Excited to announce Eterna has launched our new OpenASO project in collaboration with the Sanford and Stone Labs at UC Santa Cruz.

Our players will be designing short pieces of #RNA to restore proper splicing in pre-mRNA mutated by #genetic disease and learn more about #exon definition.

https://www.youtube.com/watch?v=Iq57MTprHV0

#chemistry #biochemistry #raredisease #hemophilia #spliceosome #crowdsourcing #citizenscience

RT @JTHjournal@twitter.com

@LiekeBaas@twitter.com et al identified that important ethical aspects of gene therapy for #hemophilia concern its potential to live up to expectations, psychosocial impacts and costs & access.
Re-thinking of the narrative of 🧬 #GeneTherapy as cure⁉️
▶️https://bit.ly/3IsEcKn

🐦🔗: https://twitter.com/JTHjournal/status/1613793601838026754

#Hemophilia is a #RareDisease, but pricing for factor 8 is symptomatic of a nearly ubiquitous problem. We, and our #Employers and #Insurers, are shopping for #Healthcare in a #Market where everything is #Monetized to the maximum without much regard for the implications for #PatientHealth.

#Healthcare #MedicareForAll #Exploitation #Unaffordable #PriceGouging

Congratulations Sean Quinn for highest scoring #ASH2022 abstract #thrombosis & haemostasis field 🏆#hemophilia 💊 treatment with Factor Va binding ab

RT @ProfMakris@twitter.com

Trevor Baglin showed the results of the use of SerpinPC (inhibitor of activated protein C) in #hemophilia at #ASH22. ABR reduced from 34 to 2.2. 100% of the patients had target joints on entry. Will the results be better in patients with better joints?

🐦🔗: https://twitter.com/ProfMakris/status/1601675762364731392

RT @pjlynch@twitter.com

Simple, digestible framing. 🙏🏽
#hemophilia
#genetherapy
#ASH22 https://twitter.com/profmakris/status/1601298977613262848

🐦🔗: https://twitter.com/pjlynch/status/1601299595253846017

"The therapy, called Hemgenix, gained US approval from the Food and Drug Administration on November 22 to treat patients with severe #hemophilia B. Shortly after its approval, CSL Behring, the pharmaceutical company commercializing the drug, announced its price: $3.5 million for a one-time dose. It’s now the most expensive drug in the world."

#GeneTherapy

https://www.wired.com/story/the-era-of-one-shot-multi-million-dollar-genetic-cures-is-here/

Great story about gene therapy cures and insurance from Wired. Will insurers pay current costs?
#genetherapy #hemophilia #AAV #medicine #Costofgoods #medmastodon #bigpharma #medicine
https://www.wired.com/story/the-era-of-one-shot-multi-million-dollar-genetic-cures-is-here/

Great story about gene therapy cures and insurance from Wired. Will insurers pay current costs?
#genetherapy #hemophilia #AAV #medicine #Costofgoods #medmastodon #bigpharma #medicine
https://www.wired.com/story/the-era-of-one-shot-multi-million-dollar-genetic-cures-is-here/

RT @KathyS_MacKay@twitter.com

WORLD AIDS DAY💔
Today, I honor those whose lives are/were impacted by HIV, including my husband, Dave, a hemophiliac. As I set the timer on my camera for this 1996 pic, I had no clue the countdown of his final year had begun. #AIDS_Day22 #hemophilia #amwriting #memoir #BigPharma

🐦🔗: https://twitter.com/KathyS_MacKay/status/1598206327352098818

Now we move into the debate of whether health insurance companies will pony up for patients to take the most expensive drug in the world. #Hemophilia #HemophiliaB #GeneTherapy

https://www.businessinsider.com/hemophilia-patients-react-to-new-3-million-gene-therapy-2022-11?fbclid=IwAR05eCU6yn549usF8Fg6wIjTtuW0W-NF0DOVT57MMrA5OkLCxsFO7kHJB4E&mibextid=Zxz2cZ

Now we move into the debate of whether health insurance companies will pony up for patients to take the most expensive drug in the world. #Hemophilia #HemophiliaB #GeneTherapy

https://www.businessinsider.com/hemophilia-patients-react-to-new-3-million-gene-therapy-2022-11?fbclid=IwAR05eCU6yn549usF8Fg6wIjTtuW0W-NF0DOVT57MMrA5OkLCxsFO7kHJB4E&mibextid=Zxz2cZ

The FDA has approved the most expensive drug in the world at $3.5m per dose. Unconscionable profiteering from Big Pharma, or cost-effective solution? (4-min read)

https://www.sciencealert.com/the-fda-just-approved-the-most-expensive-drug-in-the-world

#science #biology #DNA #geneTherapy #healthcare #health #medicine #medical #hemophilia