Hemophilia News and Research

RSS

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.

Final results from Alnylam’s ALN-TTR01 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today final results from its completed Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis.

11 May 2012

EU and U.S. regulatory authorities accept Biogen Idec’s BG-12 NDA for review

Today Biogen Idec announced that U.S. and EU regulatory authorities have accepted the company's marketing applications for the review of BG-12 (dimethyl fumarate), an oral therapeutic candidate for the treatment of multiple sclerosis.

10 May 2012

Baxter declares quarterly dividend of $0.335 per common share

The Board of Directors of Baxter International Inc. today declared a quarterly dividend of $0.335 per Baxter common share.

From Baxter International Inc. 8 May 2012

Positive results from PROLOR Biotech's hGH-CTP Phase II trial for growth hormone deficiency

PROLOR Biotech, Inc. today reported top-line results from a Phase II four-month treatment extension study of hGH-CTP, its long-acting version of human growth hormone, in growth hormone deficient adults.

7 May 2012

CSL Behring commences C1-INH phase I/II study in hereditary angioedema

CSL Behring announced today that it has initiated an international phase I/II study of a volume-reduced, subcutaneous formulation of C1-esterase inhibitor (C1-INH) concentrate in patients with hereditary angioedema.

4 May 2012

Alnylam first quarter revenues decrease to $20.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2012, and company highlights.

4 May 2012

Biogen Idec first quarter revenues increase 7% to $1.3 billion

Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing and commercialization of innovative therapies, today announced its first quarter 2012 results.

2 May 2012

Biogen Idec announces positive data from oral BG-12 second Phase 3 trial on RRMS

Today Biogen Idec announced that detailed positive data from CONFIRM, the second Phase 3 clinical trial of oral BG-12 (dimethyl fumarate) in people with relapsing-remitting multiple sclerosis (RRMS), will be presented in three platform presentations at the 64th Annual Meeting of the American Academy of Neurology (AAN) in New Orleans.

25 Apr 2012

Medgenics seeks FDA Orphan Drug Designation for INFRADURE to treat hepatitis D

Medgenics, Inc., the developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced that it has filed for Orphan Drug Designation with the U.S. Food and Drug Administration (FDA) for INFRADURE for the treatment of hepatitis D.

23 Apr 2012

NEI awards Scripps Research $10.2M grant to develop new treatments for vision disorders

A team led by a scientist from The Scripps Research Institute has been awarded a five-year, $10.2 million grant from the National Eye Institute (NEI) to develop a new type of treatment for diabetic retinopathy, macular degeneration, and other common vision disorders.

23 Apr 2012

Positive results from Alnylam’s ALN-PCS Phase I trial on severe hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive results from its Phase I clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia.

21 Apr 2012

Medgenics seeks IND approval to initiate EPODURE Biopump Phase IIb anemia study in ESRD patients

Medgenics, Inc., the developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced that it has filed an Investigational New Drug application with the U.S. Food and Drug Administration to initiate a Phase IIb multi-center, 100-patient clinical trial.

18 Apr 2012

CSL Behring commits donations to World Federation of Hemophilia GAP program

To mark World Hemophilia Day 2012, CSL Behring has again committed to making significant financial contributions and donations of clotting factor concentrate to the World Federation of Hemophilia (WFH) Global Alliance for Progress (GAP) program over the period of three years, beginning in 2012. The product donations will be made with medicine having a minimum shelf-life of one year.

17 Apr 2012

Inspiration submits IB1001 BLA with FDA for treatment of hemophilia B

Ipsen today announced that its partner, Inspiration Biopharmaceuticals, Inc., has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the approval of IB1001, an intravenous recombinant factor IX (rFIX) for the treatment and prevention of bleeding in individuals with hemophilia B.

17 Apr 2012

UM hosts symposium on next generation characterization tools for therapeutic proteins

In a concerted effort to boost regional collaboration on developing new protein drug and diagnostics, the University of Maryland (UM) School of Pharmacy has hosted the first Interdisciplinary Symposium on Next Generation Characterization Tools for Therapeutic Proteins.

16 Apr 2012

Biogen Idec launches FAMPYRA in Canada

Today, Biogen Idec Canada announces that FAMPYRA (fampridine sustained release tablets or fampridine SR) is now available for prescription for patients living with multiple sclerosis (MS) in Canada.

12 Apr 2012

Results from Abbott’s multiple interferon-free Phase 2 HCV studies to be presented at ILC 2012

Abbott will present clinical trial results from two different interferon-free, Phase 2 studies for the treatment of hepatitis C (HCV) at the International Liver Congress 2012 (ILC 2012), the annual meeting of the European Association for the Study of the Liver (EASL), April 18-22 in Barcelona, Spain.

5 Apr 2012

CSL Behring initiates rVIIa-FP Phase I study in hemophilia A and B

CSL Behring announced today the first in human dosing of recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP). The Phase I study will investigate in healthy volunteers the safety and pharmacokinetics of rVIIa-FP in comparison to placebo.

31 Mar 2012

Gene therapy improves chylomicron metabolism in LPLD patients

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene therapy Glybera (alipogene tiparvovec) is able to markedly improve chylomicron (fat particles in the blood) metabolism following consumption of a low fat meal.

29 Mar 2012

CSL Behring commences enrollment in SCIg PATH trial for CIDP

CSL Behring has announced that the first patient has been enrolled in the PATH study, an international clinical trial designed to evaluate the efficacy, safety, and tolerability of two different doses of subcutaneous immunoglobulin (SCIg), compared with placebo, in maintenance treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).

26 Mar 2012