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#sicklecelldisease — Public Fediverse posts

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

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  1. Man functionally cured of sickle cell disease through gene therapy, Farrah Reed reports. Find this article and more at canadahealthwatch.ca #SickleCellDisease #HealthBreakthrough

  2. Beam Therapeutics reveals breakthrough sickle cell treatment data at EHA2025, showcasing innovative base editing technology for potential long-term genetic therapy solutions. #Biotech #SickleCellDisease

  3. ‘Groundbreaking’ sickle cell disease treatment approved for NHS use in England
    Clinical trials find one-time gene therapy exa-cel offers ‘functional cure’ in 96.6% of patients

    theguardian.com/society/2025/j

    #142holdheap #SickleCellDisease #GeneTherapy #Medicine

  4. "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

  5. "Some stories carry echoes of 'Mississippi appendectomies' of the mid-20th century, in which Black women would go in for a different procedure and wake up to learn that their uterus had been removed."

    A STAT #investigation by Eric Boodman on how doctors are pressuring sickle cell patients into unwanted sterilizations: statnews.com/2024/05/21/sickle

    #Longreads #Health #SickleCellDisease #Journalism #Doctors

  6. "There’s this counseling, in sickle cell disease in general, when patients are doing something that a clinician doesn’t agree with. I call it the Sickle Cell Death Threat, which is like: "If you don’t do X, you are going to die.'"

    Part 1 of @STAT's series on how, for decades, doctors have pushed #SickleCellDisease patients into sterilization:

    statnews.com/2024/05/21/sickle

    #health #healthcare #medicine #reproductivehealth #SCD #eugenics #HealthEquity

  7. 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

  8. While there are numerous caveats, there are now two gene editing therapies available for people with Sickle Cell Disease, and early therapies have been very successful. It's nowhere near cheap at over $2M for one and over $3M for the other, but it's a start I suppose. Only the 1% can afford such therapy for now, but if sanity ever returns to society, I would hope that empathy will eventually win out over profits in the end. #science #crispr #genetherapy #sicklecelldisease youtu.be/uHWD8RSw4As

  9. 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

  10. 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"

  11. New gene-editing technology approved in the U-S is giving hope to millions of people worldwide suffering from sickle cell disease.The treatment for the blood...#Geneediting #Geneeditingtherapy #UK #sicklecelldisease
    Gene-editing therapy: US approves treatment for sickle cell disease
  12. 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

  13. 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

  14. 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

  15. #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

  16. 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/