Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

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

Montefiore and Einstein researchers to present data on obesity, asthma and autism at PAS 2015

Researchers from the Children's Hospital at Montefiore and Albert Einstein College of Medicine will present data from more than 50 studies that explore topics including obesity, asthma and autism at the annual meeting of the Pediatric Academic Societies.

29 Apr 2015

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals, LLC, a biopharmaceutical company dedicated to developing products for the treatment of anemias, cancers and their debilitating comorbidities, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its flagship product SANGUINATE for the treatment of Sickle Cell Disease (SCD).

8 Apr 2015

Clinical trial uses personalized cellular therapy to treat GVHD complication of bone marrow transplant

An innovative clinical trial using the science of "personalized" cellular therapy has begun enrolling children and adults suffering from graft-versus-host-disease (GVHD), a life-threatening complication of bone marrow transplantation in which donor immune lymphocytes attack the organs of the bone marrow transplant recipient.

3 Apr 2015

Emmaus Life Sciences presents Phase 3 clinical trial of PGLG treatment for sickle cell disease

Emmaus Life Sciences, Inc., a biopharmaceutical company dedicated primarily to the discovery, development and commercialization of innovative treatments and therapies for rare and orphan diseases today announced that data from the Company’s Phase 3 clinical trial of its pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell anemia and sickle beta-0 thalassemia, will be presented, during the 9th Annual Sickle Cell Disease Research and Educational Symposium and 38th National Sickle Cell Disease Scientific Meeting.

1 Apr 2015

Patients with sickle cell disease have sleep disordered breathing problem

A new study in the Journal of Clinical Sleep Medicine revealed that 44 percent of adults with sickle cell disease who report trouble sleeping actually have a clinical diagnosis of sleep disordered breathing, including sleep apnea, which lowers their oxygen levels at night.

26 Mar 2015

Mast plans to develop vepoloxamer for chronic heart failure treatment

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, arterial disease and heart failure, today announced its plans to develop vepoloxamer (MST-188) for the treatment of patients with chronic heart failure.

23 Mar 2015

MRI more accurate, safe and less painful than liver biopsy in measuring total body iron balance

Investigators at The Saban Research Institute of Children's Hospital Los Angeles have demonstrated that MR imaging of the liver is more accurate than liver biopsy in determining total body iron balance in patients with sickle cell disease and other disorders requiring blood transfusion therapy.

20 Mar 2015

New bone marrow transplant unit launched in Bangalore, India

A bone marrow transplant can mean the difference between life and death for people with blood cancers and related disorders. But many patients in India can't afford the high treatment costs, and for them a transplant is not an option. This is changing thanks to a newly launched bone marrow transplant unit at M.S. Ramaiah Medical College in Bangalore.

19 Mar 2015

Researchers use stem cells from sickle cell disease patients to grow mature red blood cells

Researchers at Johns Hopkins have successfully corrected a genetic error in stem cells from patients with sickle cell disease, and then used those cells to grow mature red blood cells, they report. The study represents an important step toward more effectively treating certain patients with sickle cell disease who need frequent blood transfusions and currently have few options.

10 Mar 2015

Loyola's Genomics Facility uses next generation sequencing to study genetic make-up of organisms

Loyola University Chicago's Genomics Facility is using an advanced technology called next generation sequencing to assist a broad range of researchers studying the genetic make-up of organisms ranging from viruses to humans.

9 Mar 2015

Investigational drug may attack cause of vaso-occlusion crises in sickle cell anemia

Treatment for painful episodes of blood vessel obstruction in sickle cell anemia is currently limited to controlling pain, but an investigational therapy might be able to interfere with the underlying cause of these events, known as vaso-occlusion crises, researchers at Duke Medicine report.

3 Mar 2015

Three Keck Medicine scientists of USC receive $4.3 million from CIRM for stem cell research

Three scientists from Keck Medicine of the University of Southern California have won grants exceeding $4.3 million from the California Institute for Regenerative Medicine for research that includes creating a temporary liver for transplant patients, finding novel ways to treat immune disorders and blood diseases and developing a new animal model for exploring diseases like heart failure, diabetes and neurodegenerative diseases.

5 Feb 2015

FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

Sangamo BioSciences, Inc. announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration and is now active.

4 Feb 2015

Johns Hopkins pediatricians offer guidelines on choosing between urgent care and ER

Parents have a natural tendency to fear the worst when it comes to their children and often opt for a "better safe than sorry" course of action, Canares says, but the truth is many situations don't warrant a trip to the emergency room. At the other end of the spectrum are cases that clearly require emergency attention but end up in urgent care instead — a less common scenario, Canares says.

2 Feb 2015

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

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

Montefiore and Einstein researchers to present data on obesity, asthma and autism at PAS 2015

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Clinical trial uses personalized cellular therapy to treat GVHD complication of bone marrow transplant

Emmaus Life Sciences presents Phase 3 clinical trial of PGLG treatment for sickle cell disease

Patients with sickle cell disease have sleep disordered breathing problem

Mast plans to develop vepoloxamer for chronic heart failure treatment

MRI more accurate, safe and less painful than liver biopsy in measuring total body iron balance

New bone marrow transplant unit launched in Bangalore, India

Researchers use stem cells from sickle cell disease patients to grow mature red blood cells

Loyola's Genomics Facility uses next generation sequencing to study genetic make-up of organisms

Investigational drug may attack cause of vaso-occlusion crises in sickle cell anemia

Three Keck Medicine scientists of USC receive $4.3 million from CIRM for stem cell research

FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

Johns Hopkins pediatricians offer guidelines on choosing between urgent care and ER

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

Sickle Cell Disease News and Research

Latest Sickle Cell Disease News and Research

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

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