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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

FGF protein may play key role in breast and prostate cancer, shows research

Simply put, cancer is caused by mutations to genes within a cell that lead to abnormal cell growth. Finding out what causes that genetic mutation has been the holy grail of medical science for decades.

13 Aug 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

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

Recently-developed drug holds great promise for children with acute lymphoblastic leukaemia

A new Australian study shows that a recently-developed drug, already used safely in adult leukaemia clinical trials, holds great promise for some children with an aggressive form of cancer known as acute lymphoblastic leukaemia (ALL).

10 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

Stem cell disease model reveals how tumor suppressor may drive bone cancer

Using induced pluripotent stem cells (iPSCs), a team led by Mount Sinai researchers has gained new insight into genetic changes that may turn a well known anti-cancer signaling gene into a driver of risk for bone cancers, where the survival rate has not improved in 40 years despite treatment advances.

10 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