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News & Perspectives

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聽SPK-9001聽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聽SPK-8011聽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.,聽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聽Sanofi聽Genzyme, the specialty care global business unit of聽Sanofi, announced Dec. 15, 2017, the聽U.S. Food and Drug Administration聽(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庐聽(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:聽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聽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鈥檝e 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聽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鈥檚 entirety, click HERE. Genentech, a member of the Roche Group, announced today positive interim results from the Phase III HAVEN 4 study evaluating HEMLIBRA庐 (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.聽 While this is true for the general population, it is especially important for individuals with hemophilia or other bleeding disorders.聽 We understand the importance that activity and a聽healthy diet play in maintaining an ideal聽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聽on December 5, 2017 outlining a voluntary recall by聽Simple Diagnostics of聽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) […]

Dear Addy: New Parent

November 27, 2017

Dear Addy, I鈥檓 a new mom and my husband and I just learned that our baby has hemophilia A. We are overjoyed at becoming parents and overwhelmed with next steps. Could you offer tips for building a relationship with a hematologist? 聽Signed, New Mom 聽Dear Mom, You鈥檝e come to the right place! Knowing where to […]

Infusing Love: A Season of Gratitude

November 22, 2017

I get some pretty stunned responses when I tell family and friends that I am grateful for hemophilia.聽 They can鈥檛 understand how it is possible that I can be thankful that my son, Thomas, has unexpected, ill-timed, and painful bleeds.聽 How could anyone find good in dealing with a nonsensical insurance company?聽 Obviously, I could […]

Genentech Releases Phase III HAVEN 3 Results

November 20, 2017

The following is an edited excerpt from a press release from Genentech. To read the press release in full, click here. Genentech, a member of the Roche Group, announced positive results from the Phase III HAVEN 3 study evaluating Hemlibra聽(emicizumab-kxwh) in adults and adolescents (aged 12 years or older) with hemophilia A without inhibitors to […]

FDA Approves Genentech’s HEMLIBRA, (Emicizumab-KXWH) for Hemophilia A with Inhibitors

November 16, 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 that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA庐聽(emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes […]

Infusing Love: Big Hearts

November 15, 2017

The past six months have been hard. Like hemophilia was managing us instead of us managing hemophilia. I can鈥檛 remember the amount of times I sobbed out of frustration, fear and being powerless to help my son. We are still muddling through and each day is a battle. A battle where it seems like we […]

Dear Addy: ABLE Accounts

November 13, 2017

Dear Addy, Are there ways for my teenage son to save money for college, without endangering his health coverage? My son is currently enrolled in Medicaid under our state鈥檚 Katie Beckett program for children with long-term disabilities. Signed, Savvy Saver Dear Saver, Here鈥檚 one option you and your son may wish to explore: opening an […]

Alnylam Announces Successful Outcome Following FDA Meeting to Discuss Fitusiran Program

November 9, 2017

The following is an edited version of a press release from Alnylam Pharmaceuticals. To read the press in full, click here. Alnylam completed a successful meeting with the U.S. Food and Drug Administration (FDA), reaching alignment on safety measures and a risk mitigation strategy for clinical studies with fitusiran.聽 These measures include protocol-specified guidelines along […]

Infusing Love: Opening Our Hearts & Home

November 9, 2017

When I got married 19 years ago, I never thought I would be a foster parent to over 40 children with special needs. It never occurred to me that I would have the blessing of being a stay-at-home mom for 14 years. I never would have thought I would end up with six forever children […]

FitFactor Video: Swimming: A Safe and Efficient Workout

November 6, 2017

Swimming is often recommended as an ideal form of exercise for people with bleeding disorders for a few different reasons. To begin with, swimming allows one to strengthen their muscles in a way that doesn鈥檛 put weight on the joints, and therefore reduces the likelihood of bleed due to impact or pressure. It also enables […]

Young Adult Podcast: Grocery Shopping on a Shoestring Budget: How to Choose Healthy Foods without Breaking the Bank

November 3, 2017

By offering additional resources and education about the benefits of self-care and exercise for young adults, we hope to inspire a lifetime of healthy choices and habits!聽Our guests on this聽podcast聽are聽Kathryn Clark, a retired dietician, and Eric Burgeson, an active young adult with hemophilia. Kathryn and Eric聽iscuss ways to keep costs low at the grocery store […]

Young Adult Podcast: Maintaining Motivation: Creating Habits for a Lifetime of Fitness

November 3, 2017

By offering additional resources and education about the benefits of self-care and exercise for young adults, we hope to inspire a lifetime of healthy choices and habits!聽Our guest on this聽podcast is聽Karen Sullivan, retired professor of Health/Fitness at Marymount University. Tune into hear Karen discuss聽how to build sustainable fitness habits and how to get back on […]

Infusing Love: Caregiver to Caregiver

November 1, 2017

If you look up National Caregiver鈥檚 month on your computer, you will find several items from the Caregivers Action Network (CAN) before anything else. If you look through the stories, tips and suggestions you will see that most of them relate to adult children caring for parents. You can find the 2016 proclamation from President […]

Dear Addy: 2018 Open Enrollment

October 30, 2017

Dear Addy, I鈥檓 applying for health insurance for the first time this open enrollment period. With a bleeding disorder, what should I look for in an insurance plan? Signed, First-time Subscriber Dear Subscriber, The聽Open Enrollment Period is a timeframe each year when health insurance plans or聽HMOs (health maintenance organizations)聽are required by law to accept applications […]

Bleeding Disorders Organizations Introduces Joint Statement Supporting Bi-partisan Alexander-Murray Legislation

October 25, 2017

Click here聽for the combined issue statement from the Coalition for Hemophilia B, the Hemophilia Federation of America, the National Hemophilia Foundation and the Hemophilia Alliance supporting bi-partisan Alexander-Murray Legislation. (Full text available in pdf and below) People with Hemophilia and Other Bleeding Disorders Urge their Senators to Support Alexander-Murray Market Stabilization Legislation Senators Lamar Alexander […]

Infusing Love: An Unexpected Lesson

October 25, 2017

I lost my job on a Monday. The following Tuesday, I was making the hour-long trek to our HTC for bi-monthly prophy for my 15 month old son, Ben. I had a lot of time to think about what had just happened, what in the world I was going to do, and where to go […]

Young Adult Podcast Series: Getting Tech-y with It: A Hemophiliacs Fitness Journey

October 19, 2017

By offering additional resources and education about the benefits of self-care and exercise for young adults, we hope to inspire a lifetime of healthy choices and habits!聽Our guest on this聽podcast is James DeFilippi, a triathlete who tells us everything that goes into his training, from his training regimen to the technology he uses to work […]


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