home.social

#sicklecelldisease — Public Fediverse posts

Live and recent posts from across the Fediverse tagged #sicklecelldisease, aggregated by home.social.

  1. europesays.com/dk/79802/ Etavopivat is the first in a new class of drugs to meet both co-primary endpoints in the phase 3 HIBISCUS trial, substantially reducing vaso-occlusive crisis events and improving haemoglobin response in sickle cell disease #Etavopivat #Haemoglobin #HIBISCUS #JacobMartin #NovoNordisk #Phase3Trial #placebo #RedBloodCells #RegulatoryApproval #SickleCellDisease #StandardOfCare #ToplineResults #VasoOcclusiveCrisis

  2. "It was at Barnes that she’d learned people with sickle cell could have children after all. As she started coming here, she’d seen other patients with kids in tow. She hadn’t thought that was possible."

    For STAT, Eric Boodman follows a #SickleCellDisease patient as she juggles pain, doubt, and the challenges of a complex disease: statnews.com/2024/11/11/coerci

    #Longreads #Health #Medicine #Sterilization

  3. Could gene therapy be a #gamechanger in the fight against #sickle-cell anemia? | DW News

    There might be fresh hope for millions of people affected by #SickleCellDisease. In the #US, a new commercially licensed gene #therapy has been administered for the first time to a patient.
    It could be a gamechanger in the fight against this disorder of unusually-shaped red #blood cells that can disrupt the victims' blood flow. This leads to severe health complications including #chronic pain and organ damage, which can also be life threatening.
    The #genetic condition is found around the world but mainly among people with West or #CentralAfrican backgrounds. According to the World Health Organization 1,000 children are born with the disease every day in Africa. And the existing treatments can be rare and expensive.

    youtube.com/watch?v=YvhidtytB5

    #SickleCellAnemia #GeneTherapy #health #news #Afrika #Afrique #Africa #إفريقيا #tv #DW

  4. New Episode: hpr4027 :: Today I learnt (2023-12-18)

    Hosted by Dave Morriss on 2024-01-09 is flagged as Explicit and is released under a CC-BY-SA license.

    Tags: #humanPhysiology, #Hemoglobin, #SickleCellDisease, #fetus, #Perl.

    hackerpublicradio.org/eps/hpr4

  5. 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 🇪🇺.

    ➡️ ema.europa.eu/en/news/first-ge

    #CHMP #PublicHealth

    🐦🔗: nitter.cz/EMA_News/status/1735

    [2023-12-15 13:56 UTC]

  6. 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 🇪🇺.

    ➡️ ema.europa.eu/en/news/first-ge

    #CHMP #PublicHealth

    🐦🔗: nitter.cz/EMA_News/status/1735

    [2023-12-15 13:56 UTC]

  7. This is seriously cool! With all the fake tech hype all around, let's not forget there is real amazing progress happening.

    #FDA Approves First #CRISPR Gene Editing Treatment for #SickleCellDisease

    scientificamerican.com/article

    "physicians remove a person’s own bone marrow #StemCells, edit them, destroy the untreated #BoneMarrow and then reinfuse the edited cells... Cas9 deactivates BCL11A in stem cells and they begin producing the fetal #hemoglobin and creating #RedBloodCells with a normal shape"

  8. FDA approves first CRISPR therapy—here’s how it works against sickle cell - Enlarge / This digitally colorized scanning electron micrograph (SEM) r... - arstechnica.com/?p=1989840 #sicklecelldisease #sicklecellanemia #hemoglobin #science #health

  9. The Food and Drug Administration on Friday approved a powerful 👉treatment for 🔸sickle cell disease🔸, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
    The therapy, called "Casgevy", from Vertex Pharmaceuticals and CRISPR Therapeutics, is the ⚠️first medicine to be approved in the United States that uses the gene-editing tool 🔹CRISPR🔹, which won its inventors the Nobel Prize in chemistry in 2020.

    The approval marks the first of two potential breakthroughs for the inherited blood disorder.

    The FDA on Friday also approved a 👉second treatment for sickle cell disease, called "Lyfgenia", a gene therapy from drugmaker Bluebird Bio.
    Both treatments work by genetically modifying a patient’s own stem cells.

    Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries the risk of rejection by the immune system, in addition to the difficult process of finding a matching donor.
    #casgevy #lyfgenia #crisper #Sicklecelldisease #sicklecell
    nbcnews.com/health/health-news

  10. The world’s 1st gene therapy for #sicklecelldisease has been approved in the UIK. Casgevy (jointly created by #CRISPRTX & #Vertex #pharma is the first #medicine licensed using the gene editing tool #CRISPR, & offers hope to 1000's as an alternative to the treatment of arduous #bone marrow #transplants.
    apnews.com/article/sickle-cell

  11. #PrimeEditing is a new #GenomeEditing technology that promises to fix #SickleCellDisease-causing mutations in a curative approach. Discover more about this technology, a collaboration of #StJude and the #BroadInstitute of MIT and #Harvard. bit.ly/3A5CbOP

  12. #PrimeEditing is a new #GenomeEditing technology that promises to fix #SickleCellDisease-causing mutations in a curative approach. Discover more about this technology, a collaboration of #StJude and the #BroadInstitute of MIT and #Harvard. bit.ly/3A5CbOP

  13. #PrimeEditing is a new #GenomeEditing technology that promises to fix #SickleCellDisease-causing mutations in a curative approach. Discover more about this technology, a collaboration of #StJude and the #BroadInstitute of MIT and #Harvard. bit.ly/3A5CbOP

  14. Nature Medicine asked 11 leading researchers for their top clinical trials to watch in 2023. The replies include #exenatide in #Parkinsonsdisease - the most prominent and promising Phase III #drugrepurposing trial in recent years -, autologous CRISPR–Cas9-modified CD34+ human stem and progenitor cells for #sicklecelldisease; and there should be more substantial data on #lecanemab for #Alzheimers disease: ncbi.nlm.nih.gov/pmc/articles/