Blood Disorder News and Research

RSS

Researchers identify enzyme as potential new drug target for blood disorders

Medical researchers have identified a key signaling protein that regulates hemoglobin production in red blood cells, offering a possible target for a future innovative drug to treat sickle cell disease.

20 Jul 2018

Mice study shows promise for treating genetic conditions during fetal development

Researchers at Carnegie Mellon University and Yale University have for the first time used a gene editing technique to successfully cure a genetic condition in a mouse model.

10 Jul 2018

FDA warns against using teething remedies for babies

According to health officials at the Food and Drug Administration (FDA), popular teething remedies for babies may not be free from dangers.

24 May 2018

Study: Most children with sickle cell anemia not getting daily antibiotics to reduce the risk

One of the greatest health threats to children with sickle cell anemia is getting a dangerous bacterial infection - but most are not receiving a key medication to reduce the risk, a new study suggests.

13 Feb 2018

Sickle cell patients, families and doctors face a ‘fight for everything’

The day before his 30th birthday, Marqus Valentine was in a panic. "I was so scared for midnight to come rolling around because subconsciously I was like, 'This is it. Tomorrow’s my last day on Earth,'" he said.

27 Dec 2017

Medicaid expansion takes a bite out of medical debt

As the Trump administration and Republicans in Congress look to scale back Medicaid, many voters and state lawmakers across the country are moving to make it bigger.

17 Nov 2017

Increasing hydroxyurea dose linked to reduction in hospitalization for young sickle cell anemia patients

St. Jude Children's Research Hospital investigators have shown that using the drug hydroxyurea to boost average fetal hemoglobin levels above 20 percent in children and teenagers with sickle cell anemia was associated with at least a two-fold reduction in hospitalization for any reason.

9 Nov 2017

FDA approves first targeted cancer treatment based on basket study

The US Food and Drug Administration has announced that it has approved the drug vemurafenib for the treatment of patients with BRAF V600-mutant Erdheim-Chester disease (ECD).

7 Nov 2017

“Chemical surgery” on embryos to free them of a gene causing thalassemia

A team of Chinese researchers have for the first time “mended” defective embryos using chemical surgery to free them of a faulty gene that leads to beta thalassemia. Until now this defect was found to be incorrigible caused due to a single misspelling in the DNA code. Beta thalassemia is a dreaded blood disorder wherein the child is unable to make healthy blood needing blood transfusions on a regular basis. The study was published this week in the journal Protein and Cell.

2 Oct 2017

New sickle cell disease poll dispels long-held perceptions of African Americans

Pfizer Inc., the National Newspaper Publishers Association, and scholars from Howard University today announced results from a new national poll designed to deepen understanding and gauge perceptions around sickle cell disease (SCD) among African Americans.

21 Sep 2017

BWH researchers discover new genetic cause for photosensitive blood disorder

Researchers from BWH have uncovered a new genetic cause for erythropoietic protoporphyria (EPP), a photosensitive blood disorder. In the past, the most well-known causes for EPP were two specific genetic disorders, but using an extended pedigree from France, the investigators found that EPP can also be caused by changes in another gene.

14 Sep 2017

Newly-discovered mutation highlights complex genetic network that underpins blood disorder

Porphyrias, a group of eight known blood disorders, affect the body's molecular machinery for making heme, which is a component of the oxygen-transporting protein, hemoglobin. When heme binds with iron, it gives blood its hallmark red color.

7 Sep 2017

Study provides insight into development of red blood cells

Every cell in the body, whether skin or muscle or brain, starts out as a generic cell that acquires its unique characteristics after undergoing a process of specialization. Nowhere is this process more dramatic than it is in red blood cells.

18 Aug 2017

Computer models provide new details of how sickle cell disease manifests inside red blood cells

Computer models developed by Brown University mathematicians show new details of what happens inside a red blood cell affected by sickle cell disease.

28 Jul 2017

US scientists make advances in modifying human embryos

American Scientists have managed to edit and improve the DNA of human embryos in an effort to correct the gene defects that cause inherited diseases.

27 Jul 2017

Latinos left out of clinical trials … and possible cures

Two decades ago, Luis Antonio Cabrera received devastating news: He likely had only three months to live.

19 Jul 2017

New treatment receives FDA approval to reduce complications of sickle cell disease

The U.S. Food and Drug Administration today approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.

7 Jul 2017

Simple treatment leads to sustained virological response in patients with thalassaemia and HCV

Many individuals with the blood disorder thalassaemia also carry the hepatitis C virus (HCV) due to contaminated transfusions before 1990.

29 Jun 2017

Finnish researchers find accumulation of gene mutations in some patients with rheumatoid arthritis

Gene mutations accumulating in cells are typical of the development of cancer. Finnish researchers found that a similar accumulation of mutations occurs also in some patients with rheumatoid arthritis.

21 Jun 2017

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo's four lead product candidates, which Sangamo expects will enter the clinic this quarter.

11 May 2017