Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice.

27 Jun 2011

EMA CHMP issues positive opinion for Baxter's IVIG therapy for multifocal motor neuropathy

Baxter International Inc. announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for extension of the therapeutic indications of KIOVIG to include a new indication for multifocal motor neuropathy (MMN), a severe, debilitating disorder requiring lifelong treatment.

From Baxter International Inc. 24 Jun 2011

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

European hematologists raise alarm on insufficient research money for blood disorders

European hematologists raise the alarm on the insufficient research money for blood disorders. "Blood cancers kill over 95,000 in the EU every year; the EU must be made aware that more research funds are needed", states Professor Robin Foa, President of the European Hematology Association (EHA) at their annual congress in London.

8 Jun 2011

Increased blood clotting tendency linked to elevated bowel cancer risk

Back in the mid 19th century, a French doctor, Armand Trousseau, discovered a connection between cancer and thrombosis - the formation of often dangerous blood clots that can lead to venous occlusion.

7 Jun 2011

Positive data from Biogen Idec's AVONEX clinical trial on multiple sclerosis presented at CMSC 2011

Biogen Idec today announced the findings from a randomized, multicenter, dose-blinded clinical trial that evaluated the effect of AVONEX dose titration, or gradual dose escalation, on flu-like symptoms associated with the therapy.

6 Jun 2011

European Commission grants Orphan Drug Designation for CSL Behring's rVIIa-FP to treat hemophilia

CSL Behring announced today that it has been granted Orphan Drug Designations by the European Commission for the development of its recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP), a novel therapy to treat hemophilia A and hemophilia B patients with inhibitors.

1 Jun 2011

Biogen Idec joins global multiple sclerosis community to celebrate third annual World MS Day

Today, Biogen Idec will join the global multiple sclerosis (MS) community in celebrating the third annual World MS Day. Biogen Idec continues to be the industry leader in researching and providing groundbreaking therapies for MS. So far in 2011, the company has brought more hope to MS patients through a number of significant milestones.

25 May 2011

Sangamo announces presentations of ZFP Therapeutics data at ASGCT 2011

Sangamo BioSciences, Inc. announced today that data from clinical, preclinical and research-stage programs focused on the development of zinc finger DNA-binding protein Therapeutics were described in twenty presentations given by Sangamo scientists and collaborators at the 14th Annual Meeting of the American Society of Gene and Cell Therapy.

23 May 2011

AMT presents long-term efficacy study of Glybera in treatment of lipoprotein lipase deficiency

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today results from a long-term efficacy study of Glybera (alipogene tiparvovec) that showed improved chylomicron metabolism could be used as a biomarker for increased lipoprotein lipase (LPL) activity in those patients missing the gene that produces this protein.

20 May 2011

CSL-Behring recombinant factor research presented

Advances that CSL Behring is making in developing innovative new technologies to meet the unmet therapeutic needs of patients with hemophilia A and B will be the topic of an oral presentation at an international medical congress on May 22, 2011. CSL Behring is a global biotherapeutics company specializing in plasma-derived and recombinant therapies and is a subsidiary of CSL Limited (ASX:CSL).

19 May 2011

Stem cells from kidneys - World first from Australian scientists

According to Australian scientists, their latest stem cell research breakthrough will bring new treatments for a growing epidemic of chronic kidney disease. They are the first in the world to generate a type of stem cell from human kidney cells. The Melbourne scientists are hoping their discovery will have far-reaching effects in treating the disease.

19 May 2011

Australian TGA approves Biogen Idec's FAMPYRA to improve walking ability in patients with MS

The Australian Therapeutic Goods Administration (TGA) has granted approval for FAMPYRA, (fampridine) 10 mg Modified Release (MR) tablet, for the symptomatic improvement of walking ability in adult patients with multiple sclerosis (MS) who have shown improvement after eight weeks of treatment.

16 May 2011

EMA agrees to Swedish Orphan Biovitrum, Biogen Idec's pediatric rFIXFc trial in hemophilia

Biogen Idec and Swedish Orphan Biovitrum today announced that the European Medicines Agency's Pediatric Committee has adopted an opinion agreeing to the pediatric investigational plan for the companies' long-lasting, fully-recombinant Factor IX Fc fusion protein.

9 May 2011

Hemophilia News and Research

Further Reading

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

EMA CHMP issues positive opinion for Baxter's IVIG therapy for multifocal motor neuropathy

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

European hematologists raise alarm on insufficient research money for blood disorders

Increased blood clotting tendency linked to elevated bowel cancer risk

Positive data from Biogen Idec's AVONEX clinical trial on multiple sclerosis presented at CMSC 2011

European Commission grants Orphan Drug Designation for CSL Behring's rVIIa-FP to treat hemophilia

Biogen Idec joins global multiple sclerosis community to celebrate third annual World MS Day

Sangamo announces presentations of ZFP Therapeutics data at ASGCT 2011

AMT presents long-term efficacy study of Glybera in treatment of lipoprotein lipase deficiency

CSL-Behring recombinant factor research presented

Stem cells from kidneys - World first from Australian scientists

Australian TGA approves Biogen Idec's FAMPYRA to improve walking ability in patients with MS

EMA agrees to Swedish Orphan Biovitrum, Biogen Idec's pediatric rFIXFc trial in hemophilia

Ipsen first quarter sales increase 6.1% to €285.8 million

Grant Thornton Leader & Innovator of the Year award for Diplomat Specialty Pharmacy president

Bayer expands sponsorship agreement with World Federation of Hemophilia

GHRI founder earns Baxter-AUPHA recognition for Health Services Research

FDA approves Bayer's new Kogenate FS storage option for hemophilia A treatment

Thought leaders gather to examine challenges facing patients with primary immunodeficiencies

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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