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.

AMT initiates dosing in Phase I/II gene therapy clinical trial for hemophilia B

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the first patient has been dosed in the Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B, a seriously debilitating and potentially lethal disease.

10 Mar 2010

Senators to FDA: Lift ban on gays donating blood

The Associated Press: "The time has come to change a policy that imposes a lifetime ban on donating blood for any man who has had gay sex since 1977, 18 senators said Thursday.

5 Mar 2010

Recipients for 2010 Scholar Awards announced

The American Society of Hematology announces the 2010 recipients of its Scholar Awards. The program is designed to support hematologists who have chosen a career in research by providing partial salary or other support during that critical period required for completion of training and achievement of status as an independent investigator.

5 Mar 2010

FDA approves CSL Behring's Hizentra for PI

CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for HizentraTM, Immune Globulin Subcutaneous (Human), 20% Liquid, for treating patients diagnosed with primary immunodeficiency (PI). A once weekly immunoglobulin (Ig) replacement therapy, Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body.

4 Mar 2010

Diplomat Specialty Pharmacy to help patients obtain financial assistance to purchase life-saving drugs

Diplomat Specialty Pharmacy, the nation's largest privately held specialty pharmacy company, is on a mission to help patients obtain the financial assistance they need to purchase life-saving drugs for such illnesses as cancer, HIV/AIDS and Hepatitis C, as well as drugs for Multiple Sclerosis, kidney disease and other chronic conditions.

4 Mar 2010

Gene therapy for LCA clears another hurdle: Study

Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection.

4 Mar 2010

Majority of PI patients can be switched to IgPro20 without dose adjustment

Data from a European multicenter (non-IND study) showing that patients with PI who switched to IgPro20 from previously available SCIg formulations achieved comparable IgG trough levels without dosage adjustment, resulting in significantly less administration volume. The data were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in New Orleans, US.

3 Mar 2010

Baxter Healthcare's IIPV product recall of HomeChoice peritoneal dialysis cyclers classified as Class I recall

Baxter Healthcare Corporation announced today that the U.S. Food and Drug Administration (FDA) has classified Baxter’s recent Urgent Product Recall regarding Increased Intraperitoneal Volume (IIPV), or overfill of the abdominal cavity, associated with HomeChoice and HomeChoice Pro peritoneal dialysis cyclers as a Class I recall.

From Baxter International Inc. 3 Mar 2010

Novo Nordisk awarded contract by VA as primary source provider of insulin

Novo Nordisk, a global healthcare company and leader in diabetes care, announced it has been awarded a contract by the U.S. Department of Veterans Affairs as the primary source provider of insulin. The one year contract began on March 1, 2010 and the VA has the option to renew the contract annually for up to an additional four years.

From Novo Nordisk 3 Mar 2010

Greffex Group signs Strategic Alliance Framework Agreement with UPMC and ISMETT

The Greffex Group of regenerative medicine companies announces the signing of a Strategic Alliance Framework Agreement with UPMC and ISMETT, Italy.

2 Mar 2010

C1-INH replacement therapy effective for treating acute swelling attacks in HAE

C1-esterase inhibitor (C1-INH) concentrate is an effective, well-tolerated therapy that rapidly relieves acute swelling attacks and successive attacks at any body location in patients with hereditary angioedema (HAE), a rare and serious genetic disorder, according to data presented today at the 2010 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting. Additional pharmacokinetic data presented at the meeting confirms that C1-INH concentrate has a longer half-life than other treatment options for HAE, which protects patients from rebound attacks.

2 Mar 2010

Patients with HAE experience prodromal symptoms before acute HAE attacks: Survey

Patients with hereditary angioedema (HAE) experience prodromal or early warning symptoms before nearly three-quarters of their HAE attacks, according to survey findings presented today at the 2010 American Academy of Allergy, Asthma & Immunology Annual Meeting.

2 Mar 2010

GTC Biotherapeutics achieves high-level production of TG20 monoclonal antibody

GTC Biotherapeutics, Inc. announced that it has achieved high-level production of TG20; an anti-CD20 monoclonal antibody (MAb). The TG20 MAb, which is produced in the milk of transgenic goats, is being co-developed by GTC and LFB as part of the LFB-GTC joint venture.

2 Mar 2010

IgPro20 protects PI patients against infection

Data presented today suggest that 20 percent liquid formulation, Immune Globulin Subcutaneous (Human) (SCIg), or IgPro20 effectively protects patients with primary immunodeficiencies (PI) against infection. The 20 percent formulation, the highest Ig concentration currently available, was also shown to sustain serum IgG levels without causing unexpected rates of adverse events. The data were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in New Orleans, US.

2 Mar 2010

Baxter International to acquire outstanding equity of ApaTech for $330 million

Baxter International Inc., a global, diversified healthcare company, and ApaTech, a private equity-backed, U.K.-based orthobiologic products company, today announced a definitive agreement whereby Baxter will acquire all of the outstanding equity of ApaTech for total consideration of up to $330 million.

From Baxter International Inc. 2 Mar 2010

Ipsen’s 2009 performance: Azzalure, Dysport and Decapeptyl approval, Adenuric partnership, drug sales up 7.6%

The Board of Directors of Ipsen, chaired by Jean-Luc Bélingard, met on 26 February 2010 to review the Group’s results for 2009, published today.

1 Mar 2010

AMT announces full year 2009 results; reports modest decrease in operating expenses

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2009.

24 Feb 2010

NCC, Pfizer provide over 10M units of ReFacto Antihemophilic Factor to underserved hemophilia patients

The National Cancer Coalition (NCC) is pleased to announce its recent collaboration with Pfizer, Inc to provide hemophilia medicines to underserved patients in the developing world. To date, NCC has received more than 10 million international units of ReFacto® Antihemophilic Factor (Recombinant), a therapy used for the treatment and prevention of bleeding in people with hemophilia A, to assist patients who otherwise would have little opportunity for adequate treatment. The value of this donation is more than $13 million, and it will help many hundreds of hemophilia patients around the world.

23 Feb 2010

LFB Biotechnologies provides new loan financing to GTC Biotherapeutics for key strategic programs

GTC Biotherapeutics, Inc. (“GTC”, NASDAQ: GTCB) announced that it has entered into an agreement with its strategic partner, LFB Biotechnologies (Les Ulis, France), pursuant to which LFB has agreed to provide $7 million of new loan financing to GTC.

23 Feb 2010

Phase 3b ODIN study of once-daily PREZISTA in HIV-1 adults presented at CROI 2010

In a new study of treatment-experienced HIV-1-infected adults with no PREZISTA® (darunavir) resistance-associated mutations (RAMs), 72 percent of patients in the PREZISTA/ritonavir (r) (800/100 mg) once-daily arm achieved undetectable viral loads (<50 copies/mL) compared with 71 percent of patients in the PREZISTA/r (600/100 mg) twice-daily arm. The study met its primary objective of non-inferiority. These data from the Phase 3b ODIN study were presented today at CROI 2010, the 17th Conference on Retroviruses and Opportunistic Infections, in San Francisco.

18 Feb 2010

Hemophilia News and Research

Further Reading

AMT initiates dosing in Phase I/II gene therapy clinical trial for hemophilia B

Senators to FDA: Lift ban on gays donating blood

Recipients for 2010 Scholar Awards announced

FDA approves CSL Behring's Hizentra for PI

Diplomat Specialty Pharmacy to help patients obtain financial assistance to purchase life-saving drugs

Gene therapy for LCA clears another hurdle: Study

Majority of PI patients can be switched to IgPro20 without dose adjustment

Baxter Healthcare's IIPV product recall of HomeChoice peritoneal dialysis cyclers classified as Class I recall

Novo Nordisk awarded contract by VA as primary source provider of insulin

Greffex Group signs Strategic Alliance Framework Agreement with UPMC and ISMETT

C1-INH replacement therapy effective for treating acute swelling attacks in HAE

Patients with HAE experience prodromal symptoms before acute HAE attacks: Survey

GTC Biotherapeutics achieves high-level production of TG20 monoclonal antibody

IgPro20 protects PI patients against infection

Baxter International to acquire outstanding equity of ApaTech for $330 million

Ipsen’s 2009 performance: Azzalure, Dysport and Decapeptyl approval, Adenuric partnership, drug sales up 7.6%

AMT announces full year 2009 results; reports modest decrease in operating expenses

NCC, Pfizer provide over 10M units of ReFacto Antihemophilic Factor to underserved hemophilia patients

LFB Biotechnologies provides new loan financing to GTC Biotherapeutics for key strategic programs

Phase 3b ODIN study of once-daily PREZISTA in HIV-1 adults presented at CROI 2010

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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