to raise funds for a friend who suffers from beta thalassemia, i'm opening a unique category!
July 19, 2026 at 9:53 AM
to raise funds for a friend who suffers from beta thalassemia, i'm opening a unique category!
In positive non election news I just got the results from my doctor and while I do officially have beta thalassemia minor, my blood is otherwise GOOD! This is such a weight off my shoulders! I’m so happy to have good blood!! ✨🩸✨
November 5, 2024 at 10:40 PM
In positive non election news I just got the results from my doctor and while I do officially have beta thalassemia minor, my blood is otherwise GOOD! This is such a weight off my shoulders! I’m so happy to have good blood!! ✨🩸✨
~40 gene therapies are approved for rare & fatal diseases:
Pediatric neurodegenerative disorders, cancers, blood diseases.
Spinal muscular atrophy, sickle cell disease, hemophilia A, beta-thalassemia, Duchenne muscular dystrophy, multiple myeloma, B cell lymphoma, etc.
15/
Pediatric neurodegenerative disorders, cancers, blood diseases.
Spinal muscular atrophy, sickle cell disease, hemophilia A, beta-thalassemia, Duchenne muscular dystrophy, multiple myeloma, B cell lymphoma, etc.
15/
March 2, 2025 at 7:55 PM
~40 gene therapies are approved for rare & fatal diseases:
Pediatric neurodegenerative disorders, cancers, blood diseases.
Spinal muscular atrophy, sickle cell disease, hemophilia A, beta-thalassemia, Duchenne muscular dystrophy, multiple myeloma, B cell lymphoma, etc.
15/
Pediatric neurodegenerative disorders, cancers, blood diseases.
Spinal muscular atrophy, sickle cell disease, hemophilia A, beta-thalassemia, Duchenne muscular dystrophy, multiple myeloma, B cell lymphoma, etc.
15/
no beta thalassemia no alpha thalassemia no iron deficiency I’m just,,, inexplicably anemic
October 8, 2024 at 8:12 PM
no beta thalassemia no alpha thalassemia no iron deficiency I’m just,,, inexplicably anemic
These kinds of trials can be so expensive and those costs add up super quickly, so if you are able to help:
https://www.gofundme.com/f/join-norahs-fight-for-a-thalassemia-free-future
https://www.gofundme.com/f/join-norahs-fight-for-a-thalassemia-free-future
Donate to Join Norah's Fight for a Thalassemia Free Future, organized by Yoko Tomioka
My dear friend Norah has been living with Beta Thalassemia Major since c… Yoko Tomioka needs your support for Join Norah's Fight for a Thalassemia Free Future
www.gofundme.com
February 4, 2025 at 9:08 PM
These kinds of trials can be so expensive and those costs add up super quickly, so if you are able to help:
https://www.gofundme.com/f/join-norahs-fight-for-a-thalassemia-free-future
https://www.gofundme.com/f/join-norahs-fight-for-a-thalassemia-free-future
Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle cell disease and beta-thalassemia that set the stage for approved gene therapies. The treatments are not accessible to everyone, though
The scientists whose work led to a game-changing therapy for sickle cell disease worry those most vulnerable can’t access it
Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle cell disease and beta-thalassemia that set the stage for approved gene thera...
www.scientificamerican.com
May 5, 2026 at 6:05 PM
Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle cell disease and beta-thalassemia that set the stage for approved gene therapies. The treatments are not accessible to everyone, though
🙏 Deeply honored to receive the 2026 Warren Alpert Prize! This award reflects the work by so many to enable transformative therapies for sickle cell disease and thalassemia, and the power of human genetics to reveal biology and guide new treatments!
hms.harvard.edu/news/2026-wa...
hms.harvard.edu/news/2026-wa...
2026 Warren Alpert Foundation Prize Awarded for Gene-Editing Therapies
Five scientists recognized for contributions to cures for sickle cell disease and beta-thalassemia
hms.harvard.edu
June 25, 2026 at 1:14 PM
🙏 Deeply honored to receive the 2026 Warren Alpert Prize! This award reflects the work by so many to enable transformative therapies for sickle cell disease and thalassemia, and the power of human genetics to reveal biology and guide new treatments!
hms.harvard.edu/news/2026-wa...
hms.harvard.edu/news/2026-wa...
Iron deficiency can also affect hemoglobin electrophoresis by decreasing HbA2. This change mimics alpha thalassemia and masks beta thalassemia.
For this reason I typically correct iron status before sending electrophoresis.
14/n
For this reason I typically correct iron status before sending electrophoresis.
14/n
November 9, 2024 at 4:10 PM
Iron deficiency can also affect hemoglobin electrophoresis by decreasing HbA2. This change mimics alpha thalassemia and masks beta thalassemia.
For this reason I typically correct iron status before sending electrophoresis.
14/n
For this reason I typically correct iron status before sending electrophoresis.
14/n
The FDA expanded approval for Casgevy, a CRISPR gene therapy, to kids as young as 2 with sickle cell or beta thalassemia. It's about intervening earlier for these severe blood disorders.
FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease and Beta Thalassemia
The U.S. Food and Drug Administration (FDA) has granted supplemental approval for Casgevy (exagamglogene autotemcel), a CRISPR-based gene therapy, for children as young as 2 years old with sickle cell
www.latimes.com
July 5, 2026 at 8:00 AM
The FDA expanded approval for Casgevy, a CRISPR gene therapy, to kids as young as 2 with sickle cell or beta thalassemia. It's about intervening earlier for these severe blood disorders.
1/2: MSK is the first hospital in New York City to offer gene therapy for sickle cell disease and beta thalassemia through its pediatric program, MSK Kids. Learn more: bit.ly/4gUKC3a
MSK Kids: First in New York City Offering Gene Therapy for Sickle Cell Disease and Beta Thalassemia
A Q&A with Jaap-Jan Boelens, MD, PhD, Chief of the Pediatric Transplant and Cellular Therapy Service at MSK.
bit.ly
January 17, 2025 at 3:06 PM
1/2: MSK is the first hospital in New York City to offer gene therapy for sickle cell disease and beta thalassemia through its pediatric program, MSK Kids. Learn more: bit.ly/4gUKC3a
FDA Approves Sickle Cell Gene Therapy for Young Kids www.medpagetoday.com/hematologyon...
FDA Approves Sickle Cell Gene Therapy for Young Kids
Casgevy also now indicated for transfusion-dependent beta thalassemia in children 2 years and up
www.medpagetoday.com
July 2, 2026 at 8:19 PM
FDA Approves Sickle Cell Gene Therapy for Young Kids www.medpagetoday.com/hematologyon...
too tired to start a thread on beta-thalassemia but similar stuff applies as per sickle cell ie the malarial resistance
March 15, 2025 at 2:40 PM
too tired to start a thread on beta-thalassemia but similar stuff applies as per sickle cell ie the malarial resistance
FDA approved CRISPR gene therapy Casgevy for kids as young as 2 (sickle cell/beta thalassemia). This lets younger patients access a stem cell gene editing treatment aiming for a functional cure. In trials, 8 kids (5-11) with sickle cell had no severe crises for 12+ months.
July 8, 2026 at 8:04 AM
FDA approved CRISPR gene therapy Casgevy for kids as young as 2 (sickle cell/beta thalassemia). This lets younger patients access a stem cell gene editing treatment aiming for a functional cure. In trials, 8 kids (5-11) with sickle cell had no severe crises for 12+ months.
Classic morphology in this patient with beta thalassemia plus some underlying liver disease. Target cells (aka codocytes) have excessive cell membrane compared to their cell volume, so the excess "bunches up" centrally.
#hematology #PathTwitter #PathResidents #pathsky
#hematology #PathTwitter #PathResidents #pathsky
December 11, 2024 at 12:28 AM
Classic morphology in this patient with beta thalassemia plus some underlying liver disease. Target cells (aka codocytes) have excessive cell membrane compared to their cell volume, so the excess "bunches up" centrally.
#hematology #PathTwitter #PathResidents #pathsky
#hematology #PathTwitter #PathResidents #pathsky
They have cured spinal muscular atrophy! The cures for cystic fibrosis and Tay-Sachs are both in trials! They have cured hemophilia A *and* B! They have cured beta-thalassemia! There are dozens and *dozens* of diseases that we can cure with a single round of gene therapy now.
September 2, 2026 at 4:01 PM
They have cured spinal muscular atrophy! The cures for cystic fibrosis and Tay-Sachs are both in trials! They have cured hemophilia A *and* B! They have cured beta-thalassemia! There are dozens and *dozens* of diseases that we can cure with a single round of gene therapy now.
Great thread; I wish I could take iron. (Anemic AND beta thalassemia minor, so no iron for me)
November 9, 2024 at 4:03 PM
Great thread; I wish I could take iron. (Anemic AND beta thalassemia minor, so no iron for me)
FDA expanded approval for Casgevy, a CRISPR gene therapy, to kids as young as two with severe sickle cell or beta thalassemia. What's striking? In trials, all 8 children (100%) with sickle cell remained free from severe vaso-occlusive crises (painful complications) for at leas...
FDA Expands Approval of Casgevy Gene Therapy for Sickle Cell Disease and Beta Thalassemia to Young C
The U.S. Food and Drug Administration (FDA) has granted expanded approval for Casgevy (exagamglogene autotemcel), a CRISPR-based gene-editing therapy, to include children as young as two years old wit
www.latimes.com
July 5, 2026 at 2:03 AM
FDA expanded approval for Casgevy, a CRISPR gene therapy, to kids as young as two with severe sickle cell or beta thalassemia. What's striking? In trials, all 8 children (100%) with sickle cell remained free from severe vaso-occlusive crises (painful complications) for at leas...
He had the sickle cell TRAIT not sickle cell. That's not a "genetic condition" that's being the carrier of a recessive gene. It doesn't cause any symptoms in the carrier. ~10% of the AA community is a silent carrier of just the sickle trait. Thalassemia Alpha and Beta is also common.
December 11, 2024 at 4:49 PM
He had the sickle cell TRAIT not sickle cell. That's not a "genetic condition" that's being the carrier of a recessive gene. It doesn't cause any symptoms in the carrier. ~10% of the AA community is a silent carrier of just the sickle trait. Thalassemia Alpha and Beta is also common.
#GeneTherapy is shifting how #SickleCell disease, hemophilia and beta thalassemia are treated. Get everything you need to know with ASH Academy’s new course! academy.hematology.o...{&utm_source=bluesky&utm_medium=organic_social&utm_campaign=2025_edu_genetherapy
#ASHEducation @ash.edu.hematology.org
#ASHEducation @ash.edu.hematology.org
May 19, 2025 at 5:00 PM
#GeneTherapy is shifting how #SickleCell disease, hemophilia and beta thalassemia are treated. Get everything you need to know with ASH Academy’s new course! academy.hematology.o...{&utm_source=bluesky&utm_medium=organic_social&utm_campaign=2025_edu_genetherapy
#ASHEducation @ash.edu.hematology.org
#ASHEducation @ash.edu.hematology.org
First patient cured of beta thalassemia with gene therapy now lives pain-free—science at its finest. 🧬❤️
First Patient to Receive Gene Therapy 'Cure' for Beta-Thalassemia Living Pain-Free
Developed at the Children's Hospital of Philadelphia (CHOP) and called Zynteglo, the medicine uses the patient's own stem cells to cure them.
www.goodnewsnetwork.org
May 11, 2025 at 12:00 AM
First patient cured of beta thalassemia with gene therapy now lives pain-free—science at its finest. 🧬❤️
Nicholas was diagnosed with TWO rare diseases, Beta Thalassemia and Duchenne Muscular Dystrophy. Doctors said he wouldn’t live past 18. But this fall, thanks to Medicaid, he’s headed to college.
Medicaid gives kids like Nicholas the ability to fight. Nicholas is why we fight.
youtu.be/t_3fhIVC9UE
Medicaid gives kids like Nicholas the ability to fight. Nicholas is why we fight.
youtu.be/t_3fhIVC9UE
Congresswoman Matsui: "Thanks to Medicaid, Nicholas is headed to college."
YouTube video by Rep. Doris Matsui
youtu.be
May 14, 2025 at 9:41 PM
Nicholas was diagnosed with TWO rare diseases, Beta Thalassemia and Duchenne Muscular Dystrophy. Doctors said he wouldn’t live past 18. But this fall, thanks to Medicaid, he’s headed to college.
Medicaid gives kids like Nicholas the ability to fight. Nicholas is why we fight.
youtu.be/t_3fhIVC9UE
Medicaid gives kids like Nicholas the ability to fight. Nicholas is why we fight.
youtu.be/t_3fhIVC9UE
Amazing. Lipid nanoparticles can take gene editing beyond the liver. Just don't ask how they get there. #medsky
endpts.com/vertex-signs...
endpts.com/vertex-signs...
Vertex signs in vivo gene editing delivery pact for sickle cell and thalassemia as it looks beyond Casgevy
Vertex partners with Orna Therapeutics to develop in vivo gene editing treatments for sickle cell disease and beta thalassemia using lipid nanoparticles.
endpts.com
January 7, 2025 at 1:37 PM
Amazing. Lipid nanoparticles can take gene editing beyond the liver. Just don't ask how they get there. #medsky
endpts.com/vertex-signs...
endpts.com/vertex-signs...
This is great but all I’m saying is the industry is missing the opportunity to found the CRISPR Board Authorized Clinical Outreach Network
World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia
Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.
www.biopharmadive.com
April 20, 2024 at 4:19 PM
This is great but all I’m saying is the industry is missing the opportunity to found the CRISPR Board Authorized Clinical Outreach Network
How about what IS killing me?
Hi, I'm beta thalassemia major.
Hi, I'm beta thalassemia major.
January 2, 2026 at 3:33 AM
How about what IS killing me?
Hi, I'm beta thalassemia major.
Hi, I'm beta thalassemia major.