Updates

March 5, 2021March 5, 2021
Events Raise Visibility, Support of Bleeding Disorders During Awareness Month

Activities are underway to mark Bleeding Disorders Awareness Month, set aside each March to call attention to such disorders as hemophilia and von Willebrand disease, ... Read more
March 4, 2021March 2, 2021
I’m Grateful for My Son’s First Pediatrician, Dr. Connelly

Back in Texas in good ol’ 1996, when my first son came into the world, a nice, well put-together doctor entered our room. He introduced ... Read more
March 3, 2021March 1, 2021
Teaching Young Adults About Hemophilia Is a Never-ending Process

My adult son, Julian, does a wonderful job managing his hemophilia. He infuses twice a week per doctor’s orders, and if he has a breakthrough ... Read more
March 3, 2021March 3, 2021
Experts Identify Unique Hemophilia Challenges, Propose Ways to Overcome

People with mild to moderate hemophilia, in particular women with the disease, face unique challenges, many that are specifically linked to a lack of information ... Read more
March 1, 2021March 1, 2021
Fitusiran Dosing in Trials Reduced to Lower Blood-clot Risk

Sanofi has reduced the dosing of its investigational treatment fitusiran in clinical trials to lower the risk of blood clots in people with hemophilia, according ... Read more

Columns, HemoWife - a Column by Alliah Czarielle

I Made the Mistake of Invalidating My Husband
March 1, 2021February 25, 2021
It just slipped out that one time. I wasn’t even thinking. My husband, Jared, and ...
News

FDA Partially Rescinds Indications for BeneFIX and Ixinity
February 26, 2021February 26, 2021
Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially ...
News, syndicated

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
February 25, 2021February 25, 2021
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Honoring Those Who Went Before Us
February 25, 2021February 24, 2021
At every annual event sponsored by the National Hemophilia Foundation, a particular room is dedicated ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Sharing Our Personal Stories With Lawmakers Is a Transformative Process
February 24, 2021February 23, 2021
Most people probably have a handful of good stories to tell at parties, but someone ...
News

Low Risk of Developing Inhibitors Found for Nuwiq Patients
February 24, 2021February 24, 2021
Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies ...

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Why I Don’t Hate My Dad for Giving Me Hemophilia
February 23, 2021February 19, 2021
My mom asked me last week if I ever curse my dad for giving me ...
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
February 23, 2021February 23, 2021
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place ...
Learning About Rare Diseases Broadened My Perspective on Health and Wellness
February 22, 2021February 18, 2021
Posture Exercises May Improve Joint Health in Children
February 22, 2021February 22, 2021
Efanesoctocog Alfa on Fast Track for Hemophilia A
February 19, 2021February 19, 2021
My Family’s Angel Has Left a Mark on Our Hearts
February 18, 2021February 17, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
February 18, 2021February 18, 2021
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ...
Tips for Offering Help to a Loved One During a Crisis
February 17, 2021February 15, 2021
Hemlibra Can Help Manage Bleeding in AHA, Study Finds
February 17, 2021February 17, 2021
Why I’d Take Many Small Bleeds Over a Large, Extended Injury
February 15, 2021February 11, 2021

Preventive Advate Found Better Than On-demand Treatment in Hemophilia A
February 15, 2021February 15, 2021
When given as a prophylactic, or preventive therapy, Advate worked better than on-demand treatment at ...
Nasal Desmopressin Recall Affects Availability Through 2023
February 12, 2021February 12, 2021
To Move From Hopelessness to Joy, I Had to Be Honest With Myself
February 11, 2021February 8, 2021

Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

In Caregiving, Raising the White Flag Can Be a Sign of Strength
February 10, 2021February 8, 2021
There is no shame in raising the white flag when caregiving challenges become too taxing. ...
News, syndicated

Pandemic Won’t Stop Rare Disease Day on Feb. 28
February 10, 2021February 11, 2021
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

I Have Hemophilia and Tattoos, and I Don’t Regret It
February 9, 2021February 5, 2021
A few years ago, I went to a tattoo convention. Before I could enter, I ...
News

Gene Therapy SPK-8016 Shows Early Promise for Severe Hemophilia A
February 8, 2021February 11, 2021
A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in ...
Columns, HemoWife - a Column by Alliah Czarielle

Why I Don’t Always Think About Hemophilia
February 8, 2021February 4, 2021
When I tell people I’m married to someone with hemophilia, I get different reactions. Some ...
News

MarzAA Shows Promise in Hemophilia Animal Models
February 5, 2021February 5, 2021
The investigational medication marzeptacog alfa activated (MarzAA) showed promise at stopping bleeding in animal models ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

As a Parent, I Can Only Show Up With What I Know
February 4, 2021February 2, 2021
One thing that struck me over the head when I became a parent was an ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Let’s End the Game of Comparisons With a Little Love
February 3, 2021February 1, 2021
Rap sessions are common at bleeding disorder conferences. During these group discussions, people come together ...
News

Kovaltry Safe, Effective for Children With Hemophilia A in Real-world Study
February 3, 2021February 3, 2021
Kovaltry (octocog alfa) is effective and safe as a preventive treatment for children with hemophilia ...
News, syndicated

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
February 2, 2021February 2, 2021
While progress was made last year on newborn screening and other policy issues critical to ...
Columns, HemoWife - a Column by Alliah Czarielle

Exercising Helps Me Realize That Pain Is Necessary for Growth
February 1, 2021January 28, 2021
After an entire year of getting little exercise, I’ve finally decided to work out again. ...
News

Immunoadsorption May Be Useful Add-on Therapy in Acquired Hemophilia
February 1, 2021February 1, 2021
Immunoadsorption, or the selective removal of antibodies from the blood, might prove useful as an ...
News

Esperoct Highly Effective as Prophylaxis for Hemophilia A, Study Suggests
January 29, 2021January 29, 2021
Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

What Does It Mean to Be Perfect and Whole?
January 28, 2021January 25, 2021
Many years ago, friends told me about a workshop they attended, which addressed newly diagnosed ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

It’s Time to Empower My Son to Manage His Bleeding Disorder
January 27, 2021January 25, 2021
My youngest son, Caeleb, recently turned 15. A celebration with his favorite dinner accompanied by ...

January 29, 2018January 29, 2018

Europe’s CHMP Recommends Approval of Hemlibra to Treat Hemophilia A

News

Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds to ... Read more