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Hydroxyurea treatment improves pulmonary function decline in children with sickle cell disease

For the first time, researchers were able to demonstrate that children diagnosed with sickle cell disease showed improvement in lung function after treatment with hydroxyurea, a treatment that is underused despite its demonstrated benefits.

19 May 2016

Musashi-2 protein regulates function, development of blood stem cells

Researchers at McMaster University's Stem Cell and Cancer Research Institute have made significant steps forward in understanding the stem cells of the human blood system after discovering how a key protein allows for better control and regeneration of these cells.

28 Apr 2016

Key opinions leaders to discuss role of cyclophosphamide in the field of HSCT

Key opinions leaders in the field of haematopoetic stem cell transplantation (HSCT) will address the role of Cyclophosphamide, an anti-cancer chemotherapy drug, during the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) that will welcome more than 4,500 delegates in the host city of Valencia, Spain from the 3rd to the 6th of April 2016.

31 Mar 2016

Patient enrollment completed in vepoloxamer Phase 3 study for treatment of sickle cell disease

Mast Therapeutics, Inc., a biopharmaceutical company developing novel, clinical-stage therapies for sickle cell disease and heart failure today announced that it has completed patient enrollment in its Phase 3 clinical study of vepoloxamer for the treatment of patients with sickle cell disease experiencing vaso-occlusive crisis, known as the EPIC study.

22 Feb 2016

Duchenne muscular dystrophy: direct effect on muscle stem cells? An interview with Dr Rudnicki

For twenty years, it has been understood that dystrophin is expressed in differentiated muscle fibers where it is part of a protein complex that crosses the membrane and connects the extracellular matrix to the actin network inside the cell to provide structural integrity.

5 Feb 2016

Three Case Western faculty members receive funding to develop new diagnostic technologies

Three Case Western Reserve University faculty members have received funding to further develop emerging technologies aimed at malaria, cystic fibrosis, and sickle cell anemia.

29 Jan 2016

NHS Blood and Transplant now offers more detailed blood testing for patients with haemoglobinopathies

Patients with blood disorders can now get detailed blood group typing to enable better matched and potentially safer transfusions.

25 Jan 2016

UC Berkeley researchers make major improvement in CRISPR-Cas9 gene editing technology

University of California, Berkeley, researchers have made a major improvement in CRISPR-Cas9 technology that achieves an unprecedented success rate of 60 percent when replacing a short stretch of DNA with another.

21 Jan 2016

PD-L1 inhibition well-tolerated, active in metastatic RCC

The programmed death-ligand 1 inhibitor atezolizumab has a favourable toxicity profile and shows anti-tumour activity in previously treated patients with metastatic renal cell carcinoma, according to phase Ia trial findings.

19 Jan 2016

Hydroxyurea could be a viable option for children with sickle cell disease

A national sickle cell disease study involving Medical University of South Carolina researchers found that for some children with sickle cell disease, the drug hydroxyurea is as effective as blood transfusions to reduce blood flow speeds in the brain. Increased blood flows are a major risk factor for stroke in these children.

5 Jan 2016

St. Jude scientists develop interactive tool to advance understanding of mutations that fuel pediatric cancer

St. Jude Children's Research Hospital scientists have developed a web application and data set that gives researchers worldwide a powerful interactive tool to advance understanding of the mutations that lead to and fuel pediatric cancer. The freely available tool, called ProteinPaint, is described in today's issue of the scientific journal Nature Genetics.

30 Dec 2015

SCCA's Fred Hutch Bone Marrow Transplant Program recognized for outstanding survival rates

The Fred Hutch Bone Marrow Transplant Program at Seattle Cancer Care Alliance has earned recognition by the Center for International Blood and Marrow Transplant Research for outperforming its expected one-year survival rates for allogeneic transplant patients - those who receive donated adult blood-forming stem cells.

23 Dec 2015

Treatment with antiplatelet therapy does not reduce pain crises in children with sickle cell disease

Treatment with the antiplatelet agent prasugrel does not significantly reduce the rate of pain crises or severe lung complications in children with sickle cell disease, according to a report published in the New England Journal of Medicine describing one of the largest and most geographically diverse international clinical trials on sickle cell disease to date.

9 Dec 2015

New technology may facilitate early detection of sickle cell disease for infants in developing countries

Researchers from Seidman Cancer Center at University Hospitals Case Medical Center and Case Western Reserve University School of Medicine presented new research findings this weekend at the 57th Annual Meeting of the American Society of Hematology in Orlando.

8 Dec 2015

Research shows significant step toward improving outcomes, reducing treatment burden for SCD patients

Emerging therapies and promising new insights in the treatment of sickle cell disease represent a significant step toward improving outcomes and reducing the treatment burden for affected children and adults. Studies reporting these advances will be presented today at the 57th American Society of Hematology Annual Meeting and Exposition.

7 Dec 2015

Sickle cell disease treatment may help protect kidney function in patients

A drug used to treat sickle cell disease may provide an added benefit of protecting patients' kidney function, according to a study appearing in an upcoming issue of the Journal of the American Society of Nephrology.

20 Nov 2015

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

Saluting his spectacular track record to date of innovative research on red blood cell disorders and his future promise as a physician/scientist, Boston Children's Hospital has selected Vijay G. Sankaran, MD, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center to receive the Rising Star Award at the hospital's third annual Global Pediatric Innovation Summit, Taking on Tomorrow (#PedInno15).

11 Nov 2015

UConn-led researchers develop simple technique for diagnosis and monitoring of sickle cell disease

A team of UConn biomedical engineers, working with colleagues from Yale, MIT, and Harvard, has developed a simple, inexpensive, and quick technique for the diagnosis and monitoring of sickle cell disease that can be used in regions where advanced medical technology and training are scarce.

5 Nov 2015

Halozyme Therapeutics announces initiation of rivipansel Phase 1 clinical trial

Halozyme Therapeutics, Inc. today announced that the first healthy subject has been dosed in a Phase 1 clinical trial evaluating the safety, tolerability and pharmacokinetics of a subcutaneous formulation of rivipansel, a compound discovered by GlycoMimetics, Inc. and being developed by Pfizer Inc., using Halozyme's ENHANZE formulation.

30 Oct 2015

Mast Therapeutics begins vepoloxamer Phase 2 study in patients with chronic heart failure

Mast Therapeutics, Inc., a clinical-stage biopharmaceutical company leveraging its molecular adhesion and sealant technology (MAST) platform to develop novel therapies for sickle cell disease, heart failure, and stroke, announced that it has initiated its Phase 2 study of vepoloxamer for the treatment of patients with chronic heart failure.

26 Oct 2015