Hemophilia B News and Research

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Silencing galectin 1 protein shrinks liver cancer tumors in mice

Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting a specific protein using gene therapy can shrink hepatocellular carcinoma (HCC) in mice.

30 Nov 2023

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

Liver cancer study could help improve the safety of certain gene therapies for hemophilia

Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases.

7 Dec 2022

What does the future look like for bleeding disorders?

In this interview, we speak to Assad Haffar, the Medical and Humanitarian Aid Director at the World Federation of Hemophilia (WFH).

23 Nov 2021

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

GlobalData: Hematology tests can be a prognostic marker to predict severity of COVID-19 patients

With the rapid increase in the number of COVID-19 cases across India, identifying the critically ill COVID-19 patients in the early stage of infection can help in assessing the risk stratification and clinical management.

21 Jul 2020

2021 health plans granted leeway to limit consumers’ benefit from drug coupons

Without medication to manage her plaque psoriasis, Jennifer Brown's face, scalp, trunk and neck periodically become covered in painful red, flaky patches so dry they crack and bleed.

6 Jul 2020

Takeda to present data from three Hematology studies at WFH 2020

Takeda Pharmaceutical Company Limited, today announced a scientific update from the AHEAD real-world study investigating the long-term outcomes associated with ADVATE in patients with hemophilia A, presented as an oral presentation at the World Federation of Hemophilia Virtual Summit 2020.

From Takeda 17 Jun 2020

Using gene therapy to help the body to make its own cancer drugs

Imagine that we could instruct our bodies to make the drugs they need themselves. The gene technology that makes this possible is called mRNA therapy, which may become a major tool in the treatment of multiple diseases.

29 May 2020

COVID survivors’ blood plasma is a sought-after new commodity

Diana Berrent learned she had tested positive for COVID-19 on a Wednesday in mid-March. Within a day, she had received 30 emails from people urging her to donate blood.

11 May 2020

With federal nod, consumers could lose the boost they get from drug ‘coupons’

Patients who get financial help from drug companies to cover their copayments for prescription drugs could owe a bigger chunk of their costs under a proposed federal rule.

16 Apr 2020

'Living drug factories' could be used to achieve long-term treatment of chronic diseases

One promising way to treat diabetes is with transplanted islet cells that produce insulin when blood sugar levels get too low.

30 Mar 2020

FDA approves immune-modulating therapy for treatment of multiple sclerosis

The U.S. Food and Drug Administration has approved ozanimod, an immune-modulating therapy invented at Scripps Research, for the treatment of adults with relapsing forms of multiple sclerosis.

26 Mar 2020

Dog study suggests that AAV in gene therapy could induce cancer

A gene therapy study in dogs has stirred up old fears that by using AAV in gene therapy, it may be increasing cancer chances.

8 Jan 2020

Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK.

6 Jan 2020

Researchers discover new way to supercharge protein production

Medicines such as insulin for diabetes and clotting factors for hemophilia are hard to synthesize in the lab. Such drugs are based on therapeutic proteins, so scientists have engineered bacteria into tiny protein-making factories.

18 Dec 2019

Inbreeding between royalty led to facial defects

The royal dynasty that ruled huge Spanish and Austrian territories over the last century was called the Habsburgs and were famous for more than their imperial majesty. The “Habsburg jaw”, a facial condition that afflicted these kings and queens, was well known. Now a new study says that this facial dysmorphism was due to inbreeding. The study was published in the Annals of Human Biology.

2 Dec 2019

Mentorship in medical school linked to increased likelihood of fellows specializing in hematology

A survey of U.S. hematology-oncology fellows suggests medical school plays an important role in shaping their interest in pursuing careers in hematology, particularly when students are exposed to hematology and oncology as part of core clerkships in internal medicine and pediatrics.

31 Oct 2019

CDC to fund sickle cell disease surveillance

The Centers for Disease Control and Prevention (CDC) has announced a funding of $1.2 million to different states in order to collect data and information from people suffering from sickle cell disease. The program called the Sickle Cell Data Collection program or SCDC is at present functional in two states - California and Georgia. With the new funds coming in, nine states would be able to collect and send population based information on the health of persons living with sickle cell disease.

27 Sep 2019