I Cell Disease News and Research

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Researchers identify painful symptoms of sickle cell disease

Researchers at Drexel University have identified the physical forces in red blood cells and blood vessels underlying the painful symptoms of sickle cell disease.

18 Oct 2012

Haploidentical bone marrow transplants for sickle cell disease: an interview with Dr. Javier Bolaños Meade

Bone marrow transplant (also called stem cell transplant) is a medical intervention that allows a physician to deliver very high doses of chemotherapy (if needed) but more importantly, deliver a new immune system to the patient to fight a disease (such as cancer).

3 Oct 2012

Wheeze predicts sickle cell disease crisis

Wheezing is a significant indicator for morbidity in patients with sickle cell disease, US researchers believe.

26 Sep 2012

New clinical trial to evaluate DEX-M74 against hereditary inclusion body myopathy

Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM). National Institutes of Health scientists from the National Center for Advancing Translational Sciences (NCATS) and the National Human Genome Research Institute (NHGRI) will conduct the clinical trial at the NIH Clinical Center.

25 Sep 2012

Bone marrow transplants hold promise for sickle cell patients

Research shows that even partially matched bone marrow transplants are able to eliminate sickle cell disease in some patients with sickle cell anemia.

24 Sep 2012

Even partially matched bone marrow transplants can eliminate sickle cell disease in some patients

In a preliminary clinical trial, investigators at Johns Hopkins have shown that even partially matched bone marrow transplants can eliminate sickle cell disease in some patients, ridding them of painful and debilitating symptoms and the need for a lifetime of pain medications and blood transfusions.

24 Sep 2012

Partially-matched bone marrow transplants can eliminate sickle cell disease in some patients

In a preliminary clinical trial, investigators at Johns Hopkins have shown that even partially-matched bone marrow transplants can eliminate sickle cell disease in some patients, ridding them of painful and debilitating symptoms, and the need for a lifetime of pain medications and blood transfusions. The researchers say the use of such marrow could potentially help make bone marrow transplants accessible to a majority of sickle cell patients who need them.

21 Sep 2012

Partially-matched bone marrow transplants can eliminate sickle cell disease in some patients

21 Sep 2012

Enrollment complete in Gamida Cell’s NiCord Phase I/II trial for hematological malignancies

Gamida Cell, a leader in adult stem cell expansion technologies and products, announced today that it has completed enrollment for a Phase I/II clinical trial of NiCord, the company's second pipeline product.

20 Sep 2012

Emmaus Medical to complete target enrollment in L-Glutamine Phase III trial for sickle cell disease

In conjunction with Sickle Cell Awareness Month, Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine technology company, announced that its U.S. Phase III clinical trial to study L-Glutamine as a treatment for sickle cell disease is nearing target enrollment completion, with investigation now underway at more than 30 clinical study sites throughout the U.S.

14 Sep 2012

Pediatric pain: An interview with Lori Kozlowski

The pain experienced by the infants, children and adolescents in our study was the result of a broad range of medical and surgical conditions.

11 Sep 2012

Targeted intervention improves safety of MRI scans for children with sickle cell disease

Jude Children's Research Hospital reports success using a targeted educational approach to teach young patients to remain motionless during MRI scans, making the process safer.

1 Sep 2012

Researchers discover why people with sickle cell disease get milder cases of malaria

Researchers at Duke University Medical Center may finally have discovered why people with sickle cell disease get milder cases of malaria than individuals who have normal red blood cells.

16 Aug 2012

bluebird bio completes $60 million Series D financing

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.

25 Jul 2012

Emmaus receives EC Orphan Medicinal Product designation for Levoglutamide

Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine technology company, and subsidiary of Emmaus Life Sciences, Inc., today announced that the European Commission (EC) has granted Orphan Medicinal Product designation for the company's investigational drug Levoglutamide (L-glutamine) for the treatment of sickle cell disease.

18 Jul 2012

Treating Sickle Cell Disease with L-Glutamine

By focusing on how best to help patients, researchers can sometimes find solutions that are much simpler than anticipated. That has been the guiding approach to our efforts to develop a treatment for sickle cell disease.

3 Jul 2012

Abnormal bone marrow stem cells drive development of myelodysplastic syndromes

Researchers at Albert Einstein College of Medicine of Yeshiva University have found that abnormal bone marrow stem cells drive the development of myelodysplastic syndromes (MDS), serious blood diseases that are common among the elderly and that can progress to acute leukemia. The findings could lead to targeted therapies against MDS and prevent MDS-related cancers. The study is published today in the online edition of the journal Blood.

2 Jul 2012

Midwest patient receives successful stem cell transplant to cure sickle cell disease

Chicagoan Ieshea Thomas is the first Midwest patient to receive a successful stem cell transplant to cure her sickle cell disease without chemotherapy in preparation for the transplant.

19 Jun 2012

Third Rock Ventures announces formation of Global Blood Therapeutics

Third Rock Ventures, LLC today announced the formation of Global Blood Therapeutics, Inc. and its $40.7 million Series A financing of the company.

15 Jun 2012

Ischemic stroke on the rise among N.J. children

Stroke is one of the top 10 causes of death among children according to the National Stroke Association. A new study published today shows that in New Jersey, the diagnosis of ischemic stroke in children, resulting from a loss or obstruction of blood to the brain, is on the rise.

13 Jun 2012

I Cell Disease News and Research

Researchers identify painful symptoms of sickle cell disease

Haploidentical bone marrow transplants for sickle cell disease: an interview with Dr. Javier Bolaños Meade

Wheeze predicts sickle cell disease crisis

New clinical trial to evaluate DEX-M74 against hereditary inclusion body myopathy

Bone marrow transplants hold promise for sickle cell patients

Even partially matched bone marrow transplants can eliminate sickle cell disease in some patients

Partially-matched bone marrow transplants can eliminate sickle cell disease in some patients

Partially-matched bone marrow transplants can eliminate sickle cell disease in some patients

Enrollment complete in Gamida Cell’s NiCord Phase I/II trial for hematological malignancies

Emmaus Medical to complete target enrollment in L-Glutamine Phase III trial for sickle cell disease

Pediatric pain: An interview with Lori Kozlowski

Targeted intervention improves safety of MRI scans for children with sickle cell disease

Researchers discover why people with sickle cell disease get milder cases of malaria

bluebird bio completes $60 million Series D financing

Emmaus receives EC Orphan Medicinal Product designation for Levoglutamide

Treating Sickle Cell Disease with L-Glutamine

Abnormal bone marrow stem cells drive development of myelodysplastic syndromes

Midwest patient receives successful stem cell transplant to cure sickle cell disease

Third Rock Ventures announces formation of Global Blood Therapeutics

Ischemic stroke on the rise among N.J. children

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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