#HemophiliaB
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hematologyadvisor.bsky.social
After a single infusion of fidanacogene elaparvovec, a majority of patients with #hemophiliaB reported that their impression of life with #hemophilia was “greatly improved” and demonstrated gains in health-related QoL measures. From @jthjournal.bsky.social.

https://bit.ly/49kPq0D
Patients With Hemophilia B Report Improvement in QoL After Gene Therapy
A majority of patients with hemophilia B reported that their impression of life with hemophilia was “greatly improved” after gene therapy with fidanacogene elaparvovec.
bit.ly
January 13, 2026 at 5:48 PM
rarediseaseadvisor.bsky.social
Nearly all (94%) responders were able to discontinue continuous prophylactic #FIXTherapy, and exogenous FIX use dropped by 96%.

Read here: https://bit.ly/4a6LqS2

#RareDisease #Hemophilia #Hematology #Hemgenix® #HemophiliaB
Gene Therapy Shows Durable 5-Year Protection in Adults With Hemophilia B
One infusion of etranacogene dezaparvovec-drlb for hemophilia B provides lasting bleed protection, stable FIX for 5 years, and a favorable safety profile.
bit.ly
December 9, 2025 at 6:17 PM
natmed.nature.com
A landmark clinical trial of gene therapy infusion for hemophilia B revealed surprising findings—reshaping the future of genetic medicine.
Read the full Turning Point essay in Nature Medicine: https://ow.ly/RVST50XvVs0
#GeneTherapy #HemophiliaB #NatureMedicine
Turning genes into medicines - Nature Medicine
The first clinical trial of a gene therapy infusion for hemophilia B revealed unexpected findings.
ow.ly
November 21, 2025 at 5:17 PM
rarediseaseadvisor.bsky.social
#GeneTherapy with fidanacogene eleparvovec is useful for improving the overall health and functional status of patients with #HemophiliaB, in addition to elevating health-related quality of life parameters. Study in @jthjournal.bsky.social

Read more: https://bit.ly/47Orssr

#RareDisease #Hemophilia
Fidanacogene Eleparvovec Improves Clinical Parameters in Hemophilia
Gene therapy remains a promising therapeutic innovation in the management of patients diagnosed with hemophilia.
bit.ly
November 11, 2025 at 7:55 PM
d-and-d-world.bsky.social
The US FDA has expanded approval for Novo Nordisk’s Alhemo, offering a once‑daily, subcutaneous option for hemophilia A & B patients without inhibitors. In trials, it cut bleeding episodes by up to 86%.
#news #Hemophiliaashtag/HemophiliaCare" class="hover:underline text-blue-600 dark:text-sky-400 no-card-link">#HemophiliaCare #FDAApproval #Hemophilia #HemophiliaA #HemophiliaB #BleedingDisorders
Novo Nordisk’s stock rises with broad US FDA approval for Alhemo
Alhemo wins FDA nod for hemophilia A and B without inhibitors, showing up to 86% fewer bleeds in a Phase III trial.
druganddeviceworld.com
August 4, 2025 at 8:27 AM
phalguniddw.bsky.social
The US FDA has expanded approval for Novo Nordisk’s Alhemo, offering a once‑daily, subcutaneous option for hemophilia A & B patients without inhibitors. In trials, it cut bleeding episodes by up to 86%.
#news #Hemophiliaashtag/HemophiliaCare" class="hover:underline text-blue-600 dark:text-sky-400 no-card-link">#HemophiliaCare #FDAApproval #Hemophilia #HemophiliaA #HemophiliaB #BleedingDisorders
Novo Nordisk’s stock rises with broad US FDA approval for Alhemo
Alhemo wins FDA nod for hemophilia A and B without inhibitors, showing up to 86% fewer bleeds in a Phase III trial.
druganddeviceworld.com
August 4, 2025 at 8:27 AM
athnnews.bsky.social
Help us learn more about Hemophilia B!

New research study open studying the safety and effectiveness of Rebinyn® (Coagulation Factor IX (Recombinant) GlycoPEGylated).

Learn how to join today! gothemophiliab.com/hemophiliab....
ATHN Transcends Hemophilia B Research Study
Join an open-label research study on the long-term safety of nonacog beta pegol (Rebinyn®) in adults and children with hemophilia B. Learn about our confidential study requirements and how you can mak...
gothemophiliab.com
June 2, 2025 at 6:16 PM
athnnews.bsky.social
Got Hemophilia B?

If you take Coagulation Factor IX (Recombinant) GlycoPEGylated (Rebinyn®) you could join a research study looking at long-term safety and effectiveness.

Interested? Find out more: gothemophiliab.com/hemophiliab....
ATHN Transcends Hemophilia B Research Study
Join an open-label research study on the long-term safety of nonacog beta pegol (Rebinyn®) in adults and children with hemophilia B. Learn about our confidential study requirements and how you can mak...
gothemophiliab.com
May 28, 2025 at 5:46 PM
athnnews.bsky.social
Help us learn more about Hemophilia B!
New research study open studying the safety and effectiveness of Rebinyn® (Coagulation Factor IX (Recombinant) GlycoPEGylated).
Learn how to join today! gothemophiliab.com/hemophiliab....
ATHN Transcends Hemophilia B Research Study
Join an open-label research study on the long-term safety of nonacog beta pegol (Rebinyn®) in adults and children with hemophilia B. Learn about our confidential study requirements and how you can mak...
gothemophiliab.com
May 28, 2025 at 5:01 PM
lunaluvgood2020.bsky.social
2 Severe HemophiliaB patients with active inhibitors
May 27, 2025 at 7:10 PM
lunaluvgood2020.bsky.social
My Husband has an elbow bleed so far we have used 96,000 dollars of FEIBA his factor

I expect it will take a few more doses

It’s terrifying to consider us not having that medicine

Medicaid saves his life, literally

HemophiliaB patient with an active inhibitor
May 21, 2025 at 2:15 AM
fleissartwork.bsky.social
Come to Austria, Europe. Just found this new article when I googled what HemophiliaB is... nobody should die because the orange clown cuts Medicaid (imho the american health system is so cruel)
Need to translate, it's in german but interesting:
www.meduniwien.ac.at/web/ueber-un...
Neue Gentherapie für Hämophilie erstmals in Österreich eingesetzt | MedUni Wien
www.meduniwien.ac.at
May 19, 2025 at 3:02 PM
athnnews.bsky.social
gothemophiliab.com/hemophiliab....
Got Hemophilia B? If you take Coagulation Factor IX
(Recombinant) GlycoPEGylated (Rebinyn®) you could
join a research study looking at long-term safety and
effectiveness. Interested?
May 12, 2025 at 2:17 PM
athnnews.bsky.social
Are you interested in Hemophilia B research? ATHN (American Thrombosis and Hemostasis Network) has a research study about treatment and safety of Coagulation Factor IX (Recombinant) GlycoPEGylated (Rebinyn®). If you join the study, you can help us learn more! gothemophiliab.com/hemophiliab....
ATHN Transcends Hemophilia B Research Study
Join an open-label research study on the long-term safety of nonacog beta pegol (Rebinyn®) in adults and children with hemophilia B. Learn about our confidential study requirements and how you can mak...
gothemophiliab.com
May 8, 2025 at 8:04 PM
cchassociation.bsky.social
🎉 Congrats to member hospital Loma Linda University Children's Hospital for becoming the first in California to offer FDA-approved immunotherapy for Hemophilia B—giving patients a more convenient, less invasive treatment option.

🔗 bit.ly/3XRmDut
#HemophiliaB #CCHA #MedicalInnovation #PatientCare
April 28, 2025 at 4:06 PM
rnaianalyst.bsky.social
My most important take-home from long-term follow-up of Spark/Pfizer #hemophiliaB #AAV gene therapy study is that alcohol consumption leads to loss in activity (likely due to liver regeneration).

www.nejm.org/doi/pdf/10.1...
April 21, 2025 at 9:45 PM
ertrunnell.bsky.social
Researchers in Italy and China used a multitool #bioinformatic approach to identify new F9 gene mutations that cause #HemophiliaB and explored their effects on disease severity.
www.jthjournal.org/article/S153...

See also: www.jthjournal.org/article/S153...
An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B
Dissection of genotype–phenotype relationships in hemophilia B (HB) is particularly relevant for challenging (mild HB) or for HB-associated but unclassified factor (F)IX missense variants.
www.jthjournal.org
April 17, 2025 at 6:32 PM
samelsonjones.bsky.social
Thrilled to mark World #Hemophilia Day with this paper showing long-term benefits after #AAV gene therapy for #hemophiliaB

prn.to/4iraiEH

Paper at: www.nejm.org/doi/10.1056/...
Children's Hospital of Philadelphia Researchers Find Gene Therapy Shows Long-Term Benefits for Hemophilia B Patients
/PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney,...
prn.to
April 17, 2025 at 6:21 PM
checkrare.bsky.social
Steven Pipe, MD, Professor of Pediatrics and Pathology at the University of Michigan, discusses positive follow-up results in patients with hemophilia B treated with Hemgenix (etranacogene dezaparvovec-drlb).
checkrare.com/positive-fol...

#CheckRare #HemophiliaB #RareHematology #RareDisease
March 19, 2025 at 4:46 PM
rnaianalyst.bsky.social
My main concern is what is the significance of elevated liver enzymes..which in this trial often went untreated and persisted?

Does this indicate there's nothing to worry about after all? #hemophiliaB #genetherapy
February 20, 2025 at 5:52 PM
rnaianalyst.bsky.social
Maybe there is HOPE for #HemophiliaB gene therapy Hemgenix by #CSL.AX (licensed from #$QURE) maintains consistent Factor IX levels (at least the 'mean') over 4 years; after

year 1: 41.5 IU/dL
y2: 36.7
y3: 38.6
y4: 37.4

CSL also sees increased uptake.

finance.yahoo.com/news/csl-beh...
February 20, 2025 at 5:38 PM
innovativehem.bsky.social
This week at Innovative Hematology a 47-year-old man made history as the first person in Indiana to undergo gene therapy for hemophilia B.

wishtv.com/news/health-...

Special thanks to WISH-TV.

#genetherapy #hemophilia #hemophiliab #hematology
#CSL
Man first in Indiana to receive gene therapy for hemophilia B
Man becomes first in Indiana to undergo gene therapy for hemophilia B. The procedure aims to address the root cause of the disorder.
wishtv.com
December 5, 2024 at 5:31 PM
rachelbiology.bsky.social
I am beyond thrilled to share this preprint with Nicholas Popp from Doug Fowler’s lab. I can’t wait to see where MultiSTEP goes next! 🧬🔬

Take a look here! ⤵️
www.biorxiv.org/content/10.1...

#deepmutationalscan #dms #vampseq #factorix #factor9 #hemophiliab
Multiplex, multimodal mapping of variant effects in secreted proteins
bioRxiv - the preprint server for biology, operated by Cold Spring Harbor Laboratory, a research and educational institution
www.biorxiv.org
April 2, 2024 at 3:00 AM