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Thalassemia is a group of genetic blood disorders that affect approximately 1,000 individuals in the United States. The most severe of these disorders is Cooley's Anemia.

People with thalassemia have a genetic defect of their red blood cells that affects the cells' ability to produce normal hemoglobin. Red blood cells use hemoglobin to carry oxygen to tissues. As a result of the defect, most forms of thalassemia produce a chronic, lifelong anemia that begins in early childhood and often must be treated with frequent transfusions.

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

7. November 2009 00:05
Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today the company’s clinical program to treat Adrenoleukodystrophy (ALD). The company is developing a gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute of Health and Medical Research (INSERM). [More]

Gene therapy saves 2 children with adrenoleukodystrophy

6. November 2009 03:02
The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases. [More]

Interim data from Schering-Plough's narlaprevir Phase IIa study

3. November 2009 03:02
Schering-Plough Corporation today reported that interim results from an ongoing Phase IIa study of narlaprevir (SCH 900518), its investigational, once-daily protease inhibitor, demonstrated potent antiviral activity in treatment-naive patients with chronic hepatitis C virus (HCV) genotype 1. [More]

Schering-Plough receives FDA complete response letter regarding PEGINTRON

31. October 2009 03:09
Schering-Plough Corp. announced today the U.S. Food and Drug Administration (FDA) has issued a complete response letter to the company's supplemental Biologics License Application regarding PEGINTRON® (pegylated interferon alfa-2b) for the adjuvant treatment of patients with stage III malignant melanoma after complete lymphadenectomy. [More]

Progenika to provide blood group genotyping reference laboratory services to ARUP

22. October 2009 08:16
Progenika, Inc. today announced that it has entered into an agreement with ARUP Laboratories (ARUP) of Salt Lake City, UT, to provide blood group genotyping reference laboratory services to ARUP to determine patient red blood cell antigen profiles when serology has logistic and functional limitations. [More]

Schering-Plough to present data on boceprevir at the AASLD 2009 Annual Meeting

19. October 2009 08:38
Schering-Plough today announced that data on boceprevir, an investigational hepatitis C virus (HCV) protease inhibitor, will be reported in an oral presentation at the American Association for the Study of Liver Diseases (AASLD) Annual Meeting in Boston, Oct. 30-Nov. 3. [More]

Schering-Plough receives FDA recommendation approval for PEGINTRON

6. October 2009 02:15
Schering-Plough Corp. announced today that the U.S. Food and Drug Administration's (FDA) Oncologic Drugs Advisory Committee (ODAC) recommended approval by a vote of six to four for PEGINTRON® (pegylated interferon alfa-2b) in the adjuvant treatment of patients with Stage III malignant melanoma. [More]

Research to develop small-molecule drug that blocks excess iron absorption

25. September 2009 06:53
Excess iron in the body, if left unchecked, can form toxic deposits in major organs leading to serious conditions including heart failure, diabetes, liver cirrhosis, arthritis, and even infertility. Iron overload can be a consequence of a genetically mutated gene known as hereditary hemochromatosis, or a consequence of red blood cell transfusions, required as life saving treatments, for patients with diseases such as thalassemia. [More]

Vitamin K prevents age-related conditions

18. September 2009 01:54
An important analysis conducted by Children's Hospital Oakland Research Institute scientists suggests the importance of ensuring optimal dietary intakes of vitamin K to prevent age-related conditions such as bone fragility, arterial and kidney calcification, cardiovascular disease, and possibly cancer. Vitamin K is concentrated in dark green plants such as spinach or Swiss chard, and is either not present or present in only small amounts in most multivitamin pills. [More]

Olympic gold medalist Shannon Miller to campaign for Stemcyte

5. September 2009 02:42
StemCyte, Inc., one of the world's preeminent cord blood banks, today announced that Shannon Miller, a 7-time Olympic Medalist and 9-time World Medalist, has signed an endorsement partnership agreement and will appear in a series of advertising and public awareness campaigns for the company. [More]

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New treatment for vaso-occlusive crisis in sickle cell patients

4. September 2009 00:45
GlycoMimetics, Inc. (GMI), a clinical-stage biotechnology company that is developing a new class of glycobiology-based therapies for a broad range of indications, today announced that a pilot study of the company’s lead candidate, GMI-1070, is underway in sickle cell patients. The first patients in this pilot study were treated at Children’s Hospital & Research Center Oakland in California. [More]

Children's Hospital Oakland converts to Masimo Rainbow SET Pulse CO-Oximetry technology

2. September 2009 02:37
Masimo (Nasdaq: MASI), the inventor of Pulse CO-Oximetry(TM) and Measure-Through Motion and Low-Perfusion pulse oximetry, announced today that Children's Hospital & Research Center Oakland has completed its system-wide conversions to Masimo Rainbow SET Pulse CO-Oximetry technology. [More]

InterMune receives $20 million event payment from Roche for initiating the Phase 2b trial of RG7227/ ITMN-191

20. August 2009 02:54
Roche (SIX: ROG.VX; RO.S, OTCQX: RHHBY) and InterMune, Inc. (Nasdaq: ITMN) today announced that the first patient has been dosed in a Phase 2b study evaluating the hepatitis C virus (HCV) protease inhibitor, RG7227/ ITMN-191, in combination with PEGASYS(R) (pegylated interferon alfa-2a) and COPEGUS(R) (ribavirin). The study, to be conducted at 45 sites globally, will further define the safety and efficacy profile of RG7227/ ITMN-191, for a treatment duration of up to 24 weeks. Approximately 300 treatment-naive patients chronically infected with HCV genotype 1 - the most difficult to treat form of the virus - will participate. [More]

Mother's immune system may block fetal treatments for blood diseases

17. August 2009 05:42
Pediatric researchers have resolved an apparent contradiction in the field of prenatal cell transplantation - a medical approach that holds future promise in correcting sickle cell disease and other serious congenital blood disorders. In a new study in animals, the researchers showed that the mother's immune response interferes with the offspring's earlier ability to tolerate transplanted donor cells. [More]

Hospital refuses admission to young adults with thalassemia and sickle cell disease

25. May 2009 19:46
Young adults with thalassemia and sickle cell diease have been trying for up to 10 years to gain admission to the only adult hospital program in central and southwestern Toronto specialized to treat their blood disorders. Since 1999, the Toronto General Hospital (TGH) has restricted the inherited blood disorders program to 99 patients requiring blood transfusions. As a result, about 150 young adults with complex disorders receive blood transfusions at the Hospital for Sick Children but no adult services. [More]

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