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Genentech joins the World Federation of Hemophilia Humanitarian Aid Program

February 21, 2019

The following is a press release from Roche. Read the press release in its entirety here. Roche announced it has joined the World Federation of Hemophilia Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in developing countries. Together […]

Novo Nordisk Receives FDA Approval for Hemophilia A Treatment

February 20, 2019

The following is an excerpt from a press release. Read the press release in its entirety here. Denmark-based Novo Nordisk received approval from the U.S. Food and Drug Administration for a new treatment for hemophilia A. However, the newly approved medication will not be available in the United States until 2020. Novo Nordisk said the […]

Genentech’s Hemlibra Provided Sustained Bleed Control in a Study of Children with Hemophilia A with Factor VIII Inhibitors

December 20, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra庐 prophylaxis in children younger than 12 years of age with hemophilia A with factor […]

Genentech’s Hemlibra Provided Sustained Bleed Control in the Largest Pivotal Study in Children with Hemophilia A with Inhibitors

December 5, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra庐聽prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII […]

Bioverativ Data Presented at ASH Underscore Potential for Once Weekly Dosing with Sustained High Factor Levels in Hemophilia A

December 5, 2018

The following is an excerpt from a press release from Bioverativ. Read the press release in its entirety here. Bioverativ Inc.,聽a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented new data from the EXTEN-A Phase 1/2a trial of BIVV001 showing that a single 65 IU/kg dose of BIVV001 extended […]

uniQure Highlights Pipeline Expansion and Advancements in Technology at Research and Development Day

November 28, 2018

The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company鈥檚 Research & Development Day held this […]

FDA Approves Genentech’s Hemlibra

October 4, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration has approved Hemlibra庐(emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, […]

Bayer Receives FDA Approval for Jivi, New Hemophilia A Treatment With Step-Wise Prophylaxis Dosing Regimen

August 31, 2018

The following is an excerpt from a press release from Bayer. Read the press release in its entirety here.聽 Bayer announced that the U.S. Food and Drug Administration has approved Jivi庐 for the routine prophylactic treatment of hemophilia A in previously-treated adults and adolescents 12 years of age or older. The initial recommended prophylactic regimen […]

U.S. District Court in Delaware rules against Shire in preliminary injunction

August 8, 2018

On Aug. 7, 2018, the U.S. District Court in Delaware ruled against Shire in its request for a preliminary injunction against Roche AG from creating, transporting and marketing its recently-approved drug Hemlibra in the U.S., which received FDA approval to treat people with hemophilia A with inhibitors in 2017. This ruling means there are no […]

Fit Factor: Inspiring Fitness Stories to Get You Going in 2018

January 9, 2018

Below, we鈥檒l introduce you to three such people: Trevor Dunn, a young man with von Willebrand Disease, Dawn Evans, an adult woman who is asymptomatic carrier of Hemophilia A, and Barry Haarde, an adult male with severe Hemophilia A.聽 All of them have discovered a form of physical activity that is enjoyable and adaptable to […]

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

January 5, 2018

Note:聽The following is聽an excerpt聽from an article by Rare Disease Report. Read the full聽article in its entirety聽here. On Jan. 4, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment […]

BioMarin’s Investigational Gene Therapy for Hemophilia A Maintains Average Factor VIII Levels within Normal Range for over One Year

July 11, 2017

Note: The following is an edited version of a press release from BioMarin.聽Read the press release from BioMarin聽in it鈥檚 entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were聽presented […]

FDA Accepts Bioverativ’s Application for Investigational Hemophilia A Drug

June 12, 2017

Bioverativ announced on June 12, 2017 that the U.S. Food and Drug Administration (FDA) has accepted the company鈥檚 Investigational New Drug (IND) application for BIVV001 (also known as rFVIIIFc-VWF-XTEN), a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. […]

Shire’s Adynovate Receives Two New FDA Approvals for Treatment of Hemophilia A

December 29, 2016

Note: The following is edited from a press release from Shire. Read the full press release in it鈥檚 entirety聽here. Shire plc recently announced that the U.S. Food and Drug Administration (FDA) has approved Adynovate, an extended circulating half-life recombinant Factor VIII treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA […]

Octapharma Announces Two Hemophilia A Clinical Trials Utilizing Wilate

November 16, 2016

The following is excerpted from a press release from Octapharma USA. Read the press release in it’s entirety here.聽 Octapharma USA has announced that WILATE庐, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection will be utilized for two important clinical trials for hemophilia A launching in December. Octapharma USA […]

FDA Approves First Single Chain Product for Hemophilia A

May 27, 2016

Note: The below is an edited version of a press release by CSL Behring.聽The original release can be read in it’s entirety聽here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]

Emicizumab Study Results Released in New England Journal of Medicine

May 26, 2016

Note: The New England Journal of Medicine released an article outlining the results of a study of a new drug,聽Emicizumab (ACE910, Chugai Pharmaceuticals), that shows reduced bleeding in patients with severe hemophilia A. Read the full article,聽Factor VIII鈥揗imetic Function of Humanized Bispecific Antibody in Hemophilia A, in the New England Journal of Medicine. This video […]

FDA Approves Needle-Free Transfer Device for Hemophilia A Product

March 31, 2016

Note: The following in an edited press release from Kedrion Biopharma. The original release can be read in its entirety here. Kedrion Biopharma has gained approval from the U.S. Food and Drug Administration to package聽Ko膩te庐 Double Viral Inactivation (DVI) Antihemophilic Factor (human)聽with Mix2Vial鈩, a needle-free transfer device. The new packaging is designed to offer hemophilia […]

FDA Approves New Recombinant Factor Following LEOPOLD Trials

March 17, 2016

Note: The following is an edited version of a press release from Bayer. The original press release can be found here.聽 The U.S. Food and Drug Administration (FDA) has approved Bayer鈥檚 KOVALTRY庐 Antihemophilic Factor (Recombinant), an unmodified, full-length factor VIII compound for the treatment of hemophilia A in children and adults. The approval is based […]

Trial for SubQ Hemophilia Inhibitor Treatment Begins

March 14, 2016

Note: The below is an edited press release from Alnylam Pharmaceuticals, Inc. The original release can be read in its entirety聽here.聽 Alnylam Pharmaceuticals, Inc. announced on Monday, March 14, 2016, that it has initiated dosing of hemophilia patients with inhibitors in Part D of an ongoing Phase 1 clinical trial evaluating a once-monthly subcutaneous dose […]

FDA Gives Orphan Drug Designation for Hemophilia A Gene Therapy

March 1, 2016

Note: The below is an edited version of a press release by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical Inc. announced on Tuesday, March 1, that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by […]

HFA Observes February 29 As Rare Disease Day

February 23, 2016

The Hemophilia Federation of America (HFA) joins the National Organization for Rare Disorders (NORD) on February 29, and others around the world in observing Rare Disease Day. The purpose of this day is to focus attention on the needs of patients and families affected by rare diseases. In the U.S., any disease affecting fewer than […]

Community Voices: An Inhibitor On The High Seas

February 16, 2016

Justin Levesque (pictured in the orange hat), poses with the crew aboard a ship bound for Iceland.聽 __________________________________________ Justin Levesque has severe hemophilia with an inhibitor. Justin recently had an opportunity of a lifetime when he spent nine days aboard a ship to Iceland as part of an art project that he developed. In this […]

Dear Addy: Why Are Medical Alert IDs Important?

January 15, 2016

Dear Addy, Several parents that I鈥檝e met at the HTC have recommended I should get my daughter a medical ID bracelet. Why is this so important? Are there resources available to help me get her one? Signed Inquiring About IDs Dear Inquiring, For patients with serious medical conditions like a bleeding disorder, it鈥檚 important that […]

Octapharma Announces US Availability of NUWIQ

January 4, 2016

聽Note: The following is an edited press release from Octapharma USA. The original release can be read in its entirety聽here. Octapharma USA announced on Monday, January 4, 2016 that NUWIQ庐, Antihemophilic Factor (Recombinant), is now commercially available. NUWIQ庐 is indicated for the treatment and control of bleeding, perioperative (surgical) management, and routine prophylaxis to reduce […]

European Commission Grants $6M to Develop New Hemophilia A Treatment

December 29, 2015

Note: The following is an edited press release from Sernova Corp. The original release can be read in its entirety here. Sernova Corp., a clinical stage regenerative medicine company, announced on Monday, December 21, 2015, that the European Commission鈥檚 Horizon 2020 program has awarded a Euro 5.6M ($6.1M USD) grant to a consortium consisting of […]

Update #2: SIPPET Study

December 10, 2015

This past weekend, the聽American Society of Hematology (ASH), a worldwide organization of 15,000+ medical and scientific experts on blood diseases,聽held their 57th Annual Meeting to discuss new treatments and research methods. During this meeting, a group of doctors from around the globe presented the findings of the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET). […]

Phase I Study Results for Once-Monthly Subcutaneous Hemophilia Treatment

December 8, 2015

Note: The following is an edited press release from Alnylam. The original release can be read in its entirety here. Alnylam Pharmaceuticals, Inc. announced on December 7, 2015 positive results from its ongoing Phase 1 clinical study with fitusiran (fi-TOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-AT3. Fitusiran is an investigational RNAi therapeutic targeting […]

Pain and Mobility Are Major Problems for Patients with Hemophilia

December 8, 2015

Note: The following originally appeared on MDMagazine.com. New research further supports the understanding that patients with hemophilia suffer from pain and mobility issues, and it went on to uncover which joints are most greatly affected by the condition. Christine Kempton, MD, MSc, from Emory University in Georgia, and colleagues used multiple strategies to find how […]

Dear Addy: Inhibitors

November 23, 2015

Dear Addy, I’m taking my son in for his annual visit, and I saw that MASAC issued a statement about inhibitors. Should I have him checked for one? Signed, Learning All I Can Dear Learning, Inhibitors are antibodies that the immune system develops in response to a clotting factor product used to treat a person […]


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