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News & Perspectives

Consolidated Postings Regarding Shire’s Lawsuit Against Genentech/Roche

January 16, 2018

In an effort to provide the hemophilia community a centralized location to find the most up-to-date statements and press releases regarding a recent injunction Shire is seeking in its lawsuit against Genentech/Roche, HFA will publish links on this page. As a reminder, HFA will NOT engage in the dialogue around patent ownership and will not be […]

Shire Provides Statement Regarding Lawsuit

January 16, 2018

Shire has published a statement regarding the ongoing lawsuit Shire has filed against Genentech/Roche. Read the full statement in its entirety here.    

HFA and NHF Issue Joint Statement Regarding Shire Lawsuit Against Genentech/Roche

January 12, 2018

In the past few weeks, we have received many inquiries regarding the scope of the injunction Shire is seeking in its lawsuit against Genentech/Roche.  Certain information has become public during this period that enables NHF and HFA to provide some limited guidance as to the scope of the injunction that Shire has requested from the […]

Genentech Provides Statement Regarding Patient Access to Hemlibra

January 11, 2018

Genentech has published a statement regarding patient access to Hemlibra (Emicizumab-kkwh). Read the full statement in its entirety here.  

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

January 9, 2018

Below, we’ll 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.  All of them have discovered a form of physical activity that is enjoyable and adaptable to […]

PSI Files Lawsuit Against U.S. Department of Health and Human Services

January 9, 2018

On January 9th, Patient Services, Inc. (PSI) filed a lawsuit against the Federal Government alleging that a 2017 Modified Advisory Opinion from the Office of the Inspector General (OIG) violates their First Amendment rights. For more information, please see the press release from PSI.

Alnylam and Sanofi Enter into Strategic Restructuring of RNAi Therapeutics Rare Disease Alliance

January 9, 2018

Note: The following is edited from a press release from Alnylam. Read the full press release in its entirety here. Alnylam Pharmaceuticals, Inc., an RNAi therapeutics company, and Sanofi announced today a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development, and commercialization of certain products for the treatment of rare genetic diseases. Specifically, Alnylam […]

Dear Addy: Accumulator Adjuster Programs

January 8, 2018

Dear Addy, I get assistance with my out-of-pocket costs from the manufacturer of my clotting factor product. My insurer just notified me that it will no longer apply that assistance to my deductible and out-of-pocket maximums! Can they do that? Signed, Distressed Over Deductibles Dear Deductibles, You are running up against a new strategy that […]

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

January 5, 2018

Note: The following is an excerpt from an article by Rare Disease Report. Read the full article in its entirety 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 […]

Infusing Love: Port to Adulthood

January 3, 2018

I have seen signs for the last few years, but on a cold day just before Christmas, I saw my 14-year-old son not as a little boy, but as a young man. There wasn’t anything extraordinary happening in the moment. He sat at one end of our sectional couch and I was sitting on the […]

Infusing Love: Different Opinions Don’t Mean They Are Wrong

December 27, 2017

“So, he’s still wearing the helmet?” The question, posed by HTC staff at our recent annual visit gave me pause. Yes, my son is almost 5 years old and still wearing his helmet any time his feet are on the floor. I know this is a controversial topic ­ I’ve seen the comment threads on […]

Dear Addy: Public Speaking

December 25, 2017

Dear Addy, I want to participate in a legislative day, but I’m a terrible public speaker. Help! Signed, Nervous Nelly Dear Nervous, You are not alone – many people worry about public speaking! Use these tips to help you become more confident and comfortable speaking during legislative meetings. Hopefully you’ll find this advice beneficial – […]

HFA Research Presents at ASH 2017

December 21, 2017

HFA’s Research Team gave and received early an holiday gift this year: knowledge. We attended the 59th annual meeting of the American Society of Hematology (ASH) in Atlanta, Georgia, December 9-12. The unofficial song of the meeting was “Let it Snow” as that (along with the once standing Georgia Dome) is what was on the […]

Infusing Love: In Appreciation of Providers

December 20, 2017

It is safe to say that most of hemophilia has been unexpected. Even when you are planning for the unexpected you get another unexpected in its place, sort of a Murphy’s Law type deal. And yet, I am always finding things about hemophilia that I never expected from our experience. One of the most profound […]

Fit Factor: Spice-Up Your Holiday Dessert Menu

December 19, 2017

Looking for a new healthy holiday dessert? This Applesauce Spice Cake has a delicious, spicy aroma and taste. It has less sugar and fat, and more fiber than most desserts. Treat it like a coffee cake to be served for brunch or for dessert with a scoop of vanilla ice cream or a dab of […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

December 15, 2017

  Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. uniQure N.V. a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene […]

BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The data […]

Spark Therapeutics and Pfizer Announce Longer-term Preliminary Data Showing Consistent and Sustained Factor IX Levels in Hemophilia B

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. Spark Therapeutics and Pfizer Inc. announced on Dec. 11, 2017, with a cumulative follow-up of more than 13 patient years of observation, all 11 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for […]

Spark Therapeutics Presents Preliminary Data on Investigational SPK-8011 Phase 1/2 Dose-escalation Clinical Trial in Hemophilia A

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. Spark Therapeutics announced on Dec. 11, 2017, it has dosed seven participants in the Phase 1/2 clinical trial of SPK-8011 in hemophilia A. The first four participants, who have been followed at least 12 weeks post […]

Bioverativ Announces Results of Eloctate Study Showing High Bleed Protection and Target Joint Resolution

December 15, 2017

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc. and Swedish Orphan Biovitrum AB announced the results of a new, post-hoc, longitudinal analysis of the pivotal Phase 3 A-LONG study and ASPIRE long-term extension study, showing that weekly prophylactic dosing with its […]

Bioverativ Announces Findings From Novel Imaging Study Using Alprolix

December 15, 2017

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc., a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced findings from a novel imaging study investigating extravascular distribution of factor IX therapies, including its leading extended […]

FDA Lifts Clinical Hold on Fitusiran

December 15, 2017

Note: The following is edited from a press release from Alnylam Pharmaceuticals. Read the full press release in its entirety here. Alnylam Pharmaceuticals Inc., a RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced Dec. 15, 2017, the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including […]

Genentech Presents New Data on Hemlibra at ASH 2017

December 15, 2017

Note: The following is edited from a press release from Genentech. Read the full press release in its entirety here. Genentech, a member of the Roche Group, announced on Dec. 9, 2017, new data from the ongoing HEMLIBRA® (emicizumab-kxwh) clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. These data […]

Shire Files Preliminary Injunction Against Genentech/Roche

December 14, 2017

Note: The following is edited from a press release from Shire. Read the full press release in its entirety here. On December 14, 2017, Shire filed a motion for preliminary injunction against Roche subsidiaries Genentech Inc. and Chugai Pharmaceutical Co., Ltd., as part of an ongoing U.S. patent infringement lawsuit surrounding Genentech/Roche’s emicizumab, currently being marketed as […]

Infusing Love: Through the Smoke

December 13, 2017

It’s been two months since wildfires ripped through Northern California leaving a path of destruction in Sonoma and Napa counties like I’ve never seen before and leaving a scar on all of our hearts. I got to see firsthand the importance of community and was proud to see our bleeding disorder community come together to […]

Dear Addy: Take Action

December 13, 2017

Dear Addy, What is the best way for me to contact my legislators when policy issues affecting the bleeding disorders community arise? Signed, Alert Advocate Dear Advocate, HFA’s Take Action Center has all you need to contact your legislators, including talking points on issues affecting the bleeding disorders community. Watch this short video on using the […]

Genentech Announces Interim Result from Phase III HAVEN 4 Study

December 8, 2017

Note: The following is an edited excerpt from a press release from Genentech. To read the press release in it’s entirety, click HERE. Genentech, a member of the Roche Group, announced today positive interim results from the Phase III HAVEN 4 study evaluating HEMLIBRA® (emicizumab-kxwh) prophylaxis dosed once every four weeks in adults and adolescents […]

FitFactor: 10 Tips to Stay Healthy this Holiday Season

December 7, 2017

The holidays are upon us, and staying healthy during this season can be challenging.  While this is true for the general population, it is especially important for individuals with hemophilia or other bleeding disorders.  We understand the importance that activity and a healthy diet play in maintaining an ideal weight and decreasing the number of bleeding episodes […]

Infusing Love: The Thief of Joy

December 5, 2017

Some of the best sessions I have attended at conferences have been rap sessions. These sessions bring together certain sub-groups in the bleeding disorder community and allow them to have open conversations, asking questions and sharing information with each other. At one rap session, many years ago, a mom in the parents rap session, was […]

Voluntary Recall of Pharmacist Choice Alcohol Prep Pads

December 5, 2017

MedWatch, the FDA safety information and adverse event reporting program released a safety alert on December 5, 2017 outlining a voluntary recall by Simple Diagnostics of three lots of Pharmacist Choice Alcohol Prep Pads due to the lack of sterility assurance and other quality issues. The affected lots are: SD2070421201 (Exp. 12/2019) SD2070420925 (Exp. 09/2019) SD2070420601 (Exp. 12/2019) […]


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