I Cell Disease News and Research

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Critically ill children with flu-like symptoms should be treated preemptively with antiviral medications

Lessons learned from the first 13 children at Johns Hopkins Children's Center to become critically ill from the H1N1 virus show that although all patients survived, serious complications developed quickly, unpredictably, with great variations from patient to patient and with serious need for vigilant monitoring and quick treatment adjustments.

8 Jan 2010

Modified blood adult stem-cell transplant regimen reverses sickle cell disease

A modified blood adult stem-cell transplant regimen has effectively reversed sickle cell disease in 9 of 10 adults who had been severely affected by the disease, according to results of a National Institutes of Health study in the Dec. 10 issue of the New England Journal of Medicine.

13 Dec 2009

EC grants orphan drug designation for MP4CO for treating sickle cell disease

Sangart, Inc., a global biopharmaceutical company dedicated to developing life-saving medicines specifically designed to enhance the perfusion and oxygenation of ischemic (oxygen deprived) tissues through targeted oxygen and other gas delivery, today announced that the European Commission (EC) has granted orphan medicinal product designation for MP4CO for the treatment of sickle cell disease.

10 Dec 2009

Preliminary study shows 'mini' stem cell transplantation may safely reverse severe sickle cell disease

Results of a preliminary study by scientists at the National Institutes of Health and Johns Hopkins show that "mini" stem cell transplantation may safely reverse severe sickle cell disease in adults.

9 Dec 2009

Hemaquest Pharmaceuticals' HQK-1001 holds promise for sickle cell disease and beta thalassemia

HemaQuest Pharmaceuticals presented data from new preclinical studies and early results from clinical trials of its lead drug candidate, HQK-1001, in sickle cell disease and beta thalassemia at the annual meeting of the American Society of Hematology in New Orleans.

8 Dec 2009

ASH meeting highlights H1N1 pandemic threat, new trends and treatment options for children with genetic blood disorder

Sickle cell disease, a condition characterized by deformed and dysfunctional red blood cells, is one of the most common genetic blood disorders affecting millions of people around the world, including more than 70,000 Americans (National Heart, Lung, and Blood Institute.

8 Dec 2009

New genetic approach may hold promise for sickle cell disease

A new genetic approach to treating sickle cell disease is showing promising results in mice, report researchers from Children's Hospital Boston. By inactivating a gene they previously discovered to be important in the laboratory, they were able to boost production of a healthy fetal form of hemoglobin in the mice, potentially compensating for the defective adult hemoglobin that causes red blood cells to "sickle" and obstruct blood flow.

8 Dec 2009

Two abstracts describing preclinical studies with GMI-1070 selected for oral presentations

GlycoMimetics, Inc., a clinical-stage biotechnology company developing a new class of glycobiology-based therapies for a broad range of indications, today announced that two abstracts describing preclinical studies with GMI-1070 have been selected for oral presentations at the 51st Annual American Society of Hematology (ASH) meeting being held December 5-8 in New Orleans.

8 Dec 2009

H1N1 virus causes more life-threatening complications than seasonal flu

Infection with the H1N1 virus, or swine flu, causes more life-threatening complications than seasonal flu in children with sickle cell disease, according to research from Johns Hopkins Children's Center.

8 Dec 2009

Study finds substantial variation in hydroxyurea utilization for sickle cell disease

A new study by researchers from the Medical College of Wisconsin, in Milwaukee, found a substantial variation in hydroxyurea utilization for pain and other sickle cell disease complications in children.

7 Dec 2009

ASH to host annual High School Student Symposium at New Orleans Marriott

The American Society of Hematology (ASH) will host its annual High School Student Symposium at the New Orleans Marriott on Thursday, December 3, at 8:00 a.m. CST.

1 Dec 2009

New initiative launched to take care of patients with sickle cell disease

The Medical College of Georgia is leading an initiative that could result in a paradigm shift in the care of patients with sickle cell disease.

23 Nov 2009

Scientists use exome sequencing method to discover the cause of mendelian disorder

For the first time, scientists have successfully used a method called exome sequencing to quickly discover a previously unknown gene responsible for a mendelian disorder.

20 Nov 2009

Scientists closer to unraveling the mechanisms of gene regulation

A team led by Penn State's Ross Hardison, T. Ming Chu Professor of Biochemistry and Molecular Biology, has taken a large step toward unraveling how regulatory proteins control the production of gene products during development and growth.

20 Nov 2009

NIH awards $2 million grant for sickle cell disease research

Researchers at the University of Illinois at Chicago will use a $2 million federal grant to investigate why patients with sickle cell disease experience chronic pain -- and to develop drugs to treat it.

2 Nov 2009

FDA-approved drug PEG-ADA shows positive results in relieving major complication of priapism

Thousands of men are afflicted with an embarrassing and painful condition that triggers spontaneous, long-lasting erections. There are limited treatment options, but a solution could be on the way thanks to new research at The University of Texas Health Science Center at Houston.

31 Oct 2009

USC to develop stem cell-based treatment for age-related macular degeneration with CIRM grant

Physician-researchers at the University of Southern California (USC) received a nearly $16 million grant from the California Institute of Regenerative Medicine (CIRM) to fund the development of a stem cell-based treatment for age-related macular degeneration, the leading cause of vision loss and blindness among the elderly.

29 Oct 2009

National study to prevent strokes in children and adolescents with sickle cell anemia

St. Jude Children's Research Hospital investigators were recently awarded a $23 million federal grant to launch a national study of the drug hydroxyurea to prevent first strokes in children and adolescents with sickle cell anemia (SCA).

29 Oct 2009

USC researchers receive grant to develop stem cell-based treatment for age-related macular degeneration

29 Oct 2009

Adenosine deaminase enzyme therapy offers hope for priapism

For men coping with painful erections lasting for long periods of time, or priapism, new research published online in The FASEB Journal (http://www.fasebj.org) offers hope. That's because researchers from the United States and China show that the enzyme adenosine deaminase may prevent priapism from progressing to penile fibrosis, a condition associated with the build up of scar tissue and eventual impotence.

27 Oct 2009

I Cell Disease News and Research

Critically ill children with flu-like symptoms should be treated preemptively with antiviral medications

Modified blood adult stem-cell transplant regimen reverses sickle cell disease

EC grants orphan drug designation for MP4CO for treating sickle cell disease

Preliminary study shows 'mini' stem cell transplantation may safely reverse severe sickle cell disease

Hemaquest Pharmaceuticals' HQK-1001 holds promise for sickle cell disease and beta thalassemia

ASH meeting highlights H1N1 pandemic threat, new trends and treatment options for children with genetic blood disorder

New genetic approach may hold promise for sickle cell disease

Two abstracts describing preclinical studies with GMI-1070 selected for oral presentations

H1N1 virus causes more life-threatening complications than seasonal flu

Study finds substantial variation in hydroxyurea utilization for sickle cell disease

ASH to host annual High School Student Symposium at New Orleans Marriott

New initiative launched to take care of patients with sickle cell disease

Scientists use exome sequencing method to discover the cause of mendelian disorder

Scientists closer to unraveling the mechanisms of gene regulation

NIH awards $2 million grant for sickle cell disease research

FDA-approved drug PEG-ADA shows positive results in relieving major complication of priapism

USC to develop stem cell-based treatment for age-related macular degeneration with CIRM grant

National study to prevent strokes in children and adolescents with sickle cell anemia

USC researchers receive grant to develop stem cell-based treatment for age-related macular degeneration

Adenosine deaminase enzyme therapy offers hope for priapism

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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