Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Research reveals genetic variants linked to treatment-related complications in children with blood diseases

Research to be presented today at the 57th American Society of Hematology Annual Meeting and Exposition reveals genetic variants that are associated with disease severity and treatment-related complications in children with blood diseases.

7 Dec 2015

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer.

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

FDA awards research grants to boost product development for patients with rare diseases

The U.S. Food and Drug Administration today announced it has awarded 18 new research grants totaling more than $19 million to boost the development of products for patients with rare diseases, which affect the lives of nearly 30 million Americans.

11 Oct 2015

Researchers identify new gene locus that may protect children from developing malaria in Africa

Published today in Nature, the findings detail a new gene locus that can explain why, in communities where everyone is constantly exposed to malaria, some children develop severe malaria and others don't. Now, researchers can be sure that this particular stretch of our DNA plays a crucial role in the progression of the disease.

1 Oct 2015

FAU researcher finds unique way to monitor sickle cell disease

A researcher from Florida Atlantic University has come up with a unique way to monitor sickle cell disease -- a serious blood disorder -- using a smart phone. With a $166,935 grant from the National Science Foundation, E. (Sarah) Du, Ph.D., assistant professor in the Department of Ocean and Mechanical Engineering in FAU's College of Engineering and Computer Science, and principal investigator, will develop a portable smart sensor and a phone application for patients to analyze and store the results of their blood tests on a smart phone.

23 Sep 2015

BMC significantly reduces emergency wait time, improves SCD care for pediatric patients

Boston Medical Center has significantly reduced the amount of time that pediatric patients experiencing pain from sickle cell disease (SCD) wait before receiving pain medication when they come to the emergency department. The hospital's quality improvement initiative cut the wait time by more than 50 percent, from an average of 56 minutes to 23 minutes, from triage to administration of the first dose of pain medication.

21 Sep 2015

New collaboration may allow researchers to develop potential gene therapies for pediatric diseases

A new collaboration between Seattle Children's Research Institute and bluebird bio, Inc., a biotechnology company based in Cambridge, Mass. and Seattle, will allow researchers to develop potentially transformative gene therapies for severe genetic and rare pediatric diseases. The collaboration supports pediatric researchers in Seattle who will work with bluebird bio scientists and the company's gene editing technology to research potential cures for genetic pediatric diseases.

11 Sep 2015

14th APS symposium highlights role of endothelin in many biological functions

Endothelin (ET) is a peptide produced by cells in the blood vessels and has powerful vessel-constricting effects. Although endothelin is mainly associated with its role in blood pressure control and cardiovascular diseases, it continues to appear in other physiological functions and diseases.

4 Sep 2015

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Sangamo BioSciences, Inc., a leader in therapeutic genome editing, announced today that the company and its collaborator, Shire plc, have agreed to revise their January 2012 collaboration and license agreement to expedite the development of ZFP Therapeutics for hemophilia A and B and Huntington's disease.

2 Sep 2015

Penn Medicine experts say that health care innovation is about testing new ideas to promote better patient care

Health care has much to learn from innovative high-tech companies, but not in the way most people think, according to a Perspective published today in the New England Journal of Medicine and authored by innovation experts from the Perelman School of Medicine and the Wharton School at the University of Pennsylvania. Innovation, they say, can most effectively achieve meaningful outcomes by testing many new ideas quickly, cheaply, and contextually.

13 Aug 2015

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that the company has reached agreements with the FDA and EMA regarding the Phase III study design outline of NiCord.

10 Aug 2015

Wayne State's faculty selected for Aspen Health Innovators Fellowship

The Washington, D.C.-based educational and policy studies organization The Aspen Institute has selected Wayne State University School of Medicine's Patrick Hines, M.D., Ph.D., associate professor of Physiology and an assistant professor of Pediatric Critical Care Medicine, one of only 20 health care leaders in the country for the inaugural class of its Health Innovators Fellowship.

25 Jul 2015

Tackling fuel poverty can help reduce debilitating sickle cell disease, save significant money for NHS

Tackling fuel poverty in the homes of people with sickle cell disease could reduce debilitating attacks and save significant money for the NHS, according to a study by Sheffield Hallam University funded by the Chesshire Lehmann Fund.

22 Jun 2015

Mast Therapeutics initiates Phase 3 extension study of vepoloxamer in sickle cell disease

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 arterial disease, today reported that it has initiated an open-label, multicenter Phase 3 extension study of vepoloxamer (MST-188) in sickle cell disease, referred to as "EPIC-E."

26 May 2015

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy

29 Apr 2015

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Research reveals genetic variants linked to treatment-related complications in children with blood diseases

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Sickle cell disease treatment may help protect kidney function in patients

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

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

Halozyme Therapeutics announces initiation of rivipansel Phase 1 clinical trial

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

FDA awards research grants to boost product development for patients with rare diseases

Researchers identify new gene locus that may protect children from developing malaria in Africa

FAU researcher finds unique way to monitor sickle cell disease

BMC significantly reduces emergency wait time, improves SCD care for pediatric patients

New collaboration may allow researchers to develop potential gene therapies for pediatric diseases

14th APS symposium highlights role of endothelin in many biological functions

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Penn Medicine experts say that health care innovation is about testing new ideas to promote better patient care

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Wayne State's faculty selected for Aspen Health Innovators Fellowship

Tackling fuel poverty can help reduce debilitating sickle cell disease, save significant money for NHS

Mast Therapeutics initiates Phase 3 extension study of vepoloxamer in sickle cell disease

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

The future of automation: Machine learning–driven hepatocyte and organoid counting

From street to lab: Advancing controlled substance testing with handheld Raman and FTIR

Reimagining light scattering for biotherapeutics

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

The future of automation: Machine learning–driven hepatocyte and organoid counting

From street to lab: Advancing controlled substance testing with handheld Raman and FTIR

Reimagining light scattering for biotherapeutics

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