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Genentech Medical Communications Line Available to Answer Questions About Hemlibra 

March 27, 2018

HFA was informed by Genentech on March 26, 2018, that a total of five patient deaths have occurred while the patients were using Hemlibra (emicizumab-kxwh). Genentech has little information that they can presently share about the circumstances surrounding the most recent patient deaths; however, Genentech could confirm that the patients had received Hemlibra as part of compassionate use and expanded patient access. Genentech has a Medical Communications line at 1(800)-821-8590 for patients, concerned community members, and healthcare providers who seek further information. […]

Alnylam & Sanofi Genzyme Report Positive Results from Ongoing Phase 2 Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or Without Inhibitors

July 10, 2017

Note: The following is an edited version of a press release from Alnylam. Read the press release from Alnylam in it’s entirety here. Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme, announced today new positive results from the ongoing Phase 2 open-label extension study with fitusiran in patients with hemophilia A and B, with or without inhibitors. These results were presented today […]

Breaking Clinical Trial News from Genentech/Roche

February 22, 2017

The following is the full statement from Genentech/Roche regarding the recent death of a 41-year-old hemophilia with inhibitors patient who was participating in the HAVEN 1 study. Dear members of the Haemophilia community, At Roche, we respect and value the close relationships that exist within the haemophilia community. We also understand the important role patient […]

Infusing Love: A Letter to Hemophilia

January 27, 2016

Dear Hemophilia, There was a time when you devastated me. You destroyed my little boy’s knee and ankle, causing tremendous pain and forcing him to undergo several port surgeries. He missed weeks of school. Oh, and let’s not forget the wheelchair you put him in for over a year. That was torture, taking away his […]

Infusing Love: Timeline of a January Bleed

January 6, 2016

Thomas has had his inhibitor for more than a decade, causing bleeds to be an unfortunate, but relatively routine, occurrence. We’re used to holiday bleeds, birthday bleeds, and random “where did that come from” bleeds. Be it crass or an obvious statement, bleeding happens, and we push forward. Through these tough times, we try to […]

Update #2: SIPPET Study

December 10, 2015

This past weekend, the American Society of Hematology (ASH), a worldwide organization of 15,000+ medical and scientific experts on blood diseases, 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). […]

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 […]

FDA Grants Breakthrough Therapy for Subcutaneous Inhibitor Treatment

September 4, 2015

Note: This is an edited form of a press release from Genentech. To read the original release in its entirety, click here. Genentech announced on Friday, September 4, 2015, that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to ACE910 (RG6013, RO5534262) for the prophylactic treatment of people who are 12 years […]

FDA Grants Orphan Status for New Inhibitor Treatment

July 2, 2015

Below is from a press release Apitope sent out on June 9, 2015. To read it in its entirety, click here. Apitope, the drug discovery and development company focused on disease-modifying treatments that reinstate immune tolerance, announced today that pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the US Food and Drug […]

How a Second Grader with Hemophilia Copes with Pain

June 9, 2015

Kelly and Brian of Oregon never expected their son, Bubba, to have severe hemophilia A, and they certainly never expected Bubba to experience the complications of an inhibitor and chronic pain in his childhood. Kelly shared her family’s experiences with HFA with hopes that other families can learn from their experiences. How did you find […]

New Study Deems Long Lasting Anti-Hemophilia Factor Safe in Kids

April 27, 2015

Note: This press release was originally published by Children’s Hospital Los Angeles. To read the original post, please click here. ____________________ Children with hemophilia A require three to four infusions each week to prevent bleeding episodes, chronic pain and joint damage. The effect on quality of life can be significant, due to time and discomfort […]

Five New Developments in Hemophilia

April 7, 2015

This post first appeared on Vector, a blog of Boston Children’s Hospital an was written by Ellis Neufeld, MD, PhD, a hematologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.   ________________________________________________ From new longer-acting drugs to promising gene therapy trials, much is changing in the treatment of hemophilia, the inherited bleeding disorder in which the blood does not clot. […]

Inhibitor Patients Have Increased Risk for Joint Disease & Other Complications

April 3, 2015

Note: The below email was sent by the Centers Disease Control and Prevention (CDC) on April 3, 2015: People with hemophilia lack a protein (a clotting factor) necessary for blood to clot normally. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. The best way to treat hemophilia is to replace the […]

Baxter Sees Promising Results in Inhibitor Treatment Trial

March 13, 2015

Baxter International Inc. announced positive results from its Phase III clinical trial evaluating the safety and efficacy of BAX 817, an investigational recombinant factor VIIa (rFVIIa) treatment for people with Hemophilia A or B who develop inhibitors. The prospective, open-label, randomized, multicenter trial was designed to assess the safety and efficacy of BAX 817 in […]

Update #3: NHF’s MASAC Assessment on Inhibitor Studies

December 3, 2014

On September 24, 2014, the medical journal, Blood, came out with a study called, Recombinant Factor VIII Products and Inhibitor Development in Previously Untreated Boys with Severe Hemophilia A.  This study suggests that inhibitor incidence is higher in previously untreated patients (PUPs) with severe hemophilia A that are using Kogenate/Helixate.  The World Hemophilia Federation (WHF) issued a statement on October 6, and another […]

Update #2: FDA will Review Study Suggesting Higher Inhibitor Incidence in PUPS

November 20, 2014

On September 24, 2014, the medical journal, Blood, came out with a study called, Recombinant Factor VIII Products and Inhibitor Development in Previously Untreated Boys with Severe Hemophilia A.  This study suggests that inhibitor incidence is higher in previously untreated patients (PUPs) with severe hemophilia A that are using Kogenate/Helixate. The Food and Drug Administration (FDA) and European Medicines Agency (EMA) have […]

An Inhibitor Mom Shares Her Story With the FDA

September 25, 2014

On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA. HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out […]

Leveling with the FDA About Hemophilia Inhibitors

September 23, 2014

On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA. HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out to […]

HFA’s Excutive Director to FDA: Treatment is Not One Size Fits All

September 23, 2014

On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA. HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out to share […]

Researchers Prevent Inhibitors in Mice with Hemophilia

September 5, 2014

New and exciting news recently published in the journal Blood about a possible method to prevent and treat inhibitors. A University of Pennsylvania researcher has successfully prevented inhibitors from forming in mice with hemophilia by using a plant-based drug to teach the immune system to tolerate clotting factors. Read this University of Pennsylvania  press release in its entirety. ____________________________ […]

Journal of Blood Medicine: Inhibitor Challenges

August 26, 2014

Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Approximately 30% of people with severe Hemophilia A are affected by inhibitors at some […]

Penn Research Could Improve Hemophilia Treatment

July 17, 2014

*This article was originally posted on July 17, 2014 in Penn Current and was written by Katherine Unger Baillie. Click here to read this article in its entirety. ____________________________________ Hemophilia is a rare but potentially dangerous disease. People with the condition produce very low levels of clotting factor, the proteins in blood that stop bleeding and help begin the […]

We Did Not Fail Treatment, It Failed Us

May 20, 2014

When my son Matthew was born with severe Hemophilia A in 1994, I thought I was prepared. I had been raised by a Dad with hemophilia, lost him to HIV in 1986, and had thoroughly researched product safety prior to deciding to give birth to my son. I had prenatal testing done and knew that […]

CDC Six-Year Inhibitor Study Released

May 16, 2014

The journal Haemophilia has published the results of a six-year study called the Hemophilia Inhibitor Research Study (HIRS) that was designed to test the feasibility of conducting national monitoring for inhibitors among people with hemophilia in the United States. The study collected blood specimens on a regular basis from study participants, which were tested at […]

CDC Webinar: New Challenges in Hemophilia

April 8, 2014

On Thursday, April 3, 2014 the Centers for Disease (CDC) control hosted an informative and important webinar about new challenges facing the hemophilia community. If you missed it, take a look at Dr. Guy Young, Director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles, presentation slides about about the impact of: overweight and […]

(You Gotta) Fight for Your Right (to Party)

January 8, 2014

Nora unknowingly opened a floodgate when she did not share some candy with Thomas one afternoon. He got super moody. I left it alone for a bit, but I could hear him trying not to cry in the backseat of the car. When we pulled in the driveway, I sent the girls inside and asked […]

Changes in Latitude of My Attitude & Gratitude

November 27, 2013

I think Thanksgiving gets more hype than it should. It starts at an early age – we encourage preschoolers to make construction paper turkeys from their hands and write what they are thankful for on the “feathers.” Elementary and middle school students express their gratitude in the form of coat or canned food drives at […]

The Fighter

July 31, 2013

By Sonji Wilkes Several summers ago, I signed the kids up for swim lessons to help keep them busy during summer break. A couple of years later, after once-a-week, year-round lessons, they had progressed to a point where I was brave enough to sign them up for a local swim team.  I was thrilled; they […]

That Almost Never Happens

March 6, 2013

                At the very first meeting we had at the Hemophilia Treatment Center (HTC) when Thomas was a week old, Nathan asked the HTC team, “Does the medicine ever stop working? Can you build up antibodies to it?” Not wanting to worry us and having a good track […]


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