Updates

March 18, 2019March 18, 2019
Prophylaxis Partially Prevents Bleeding in Severe Hemophilia A Patients, Real‐world Study Finds

Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and adult males with severe hemophilia A and without inhibitors, ... Read more
March 15, 2019March 15, 2019
Hemlibra Approved in Europe to Prevent Bleeds in Hemophilia A Patients Without Factor VIII Inhibitors

People with severe hemophilia A living in Europe can now receive treatment with Hemlibra (emicizumab-kxwh) to prevent bleeding episodes. The European Commission approved ... Read more
March 14, 2019March 13, 2019
Ready, Set … Off to the Hospital?!

So, we are in the car. Everyone is in their respective seats. We are all looking forward to our vacation. The car is filled ... Read more
March 14, 2019March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more
March 13, 2019March 12, 2019
The Struggles of Dating with a Chronic Illness

Looking at myself now, my younger self never would have expected me to be where I am. Recalling my younger years, I remember having ... Read more

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

Bleeding Disorders Awareness Month: Being Present
March 13, 2019March 12, 2019
“I’m so sorry. What can I do? I am here if you need me.” ...
News

Many Adults, Families Affected by Hemophilia Support Preconception, Prenatal Genetic Screens, Study Finds
March 13, 2019March 13, 2019
More than half of adults who either have hemophilia or are family members affected ...
News

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 13, 2019March 13, 2019
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ...
News

Erectile Dysfunction More Prevalent with Age in Men With Hemophilia, Study Says
March 11, 2019March 11, 2019
Erectile dysfunction, more commonly known as male impotence, is associated with older age in ...
News

Advate is Safe, Effective Therapy for Hemophilia A, Japanese Study Finds
March 8, 2019March 8, 2019
Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

The Unique MacDonald Family Vocabulary
March 7, 2019March 6, 2019
Every family speaks their own language. Mine certainly does. It is like a secret ...

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Bleeding Disorder Awareness Month: The Hemophilia Holocaust
March 6, 2019March 4, 2019
The 1980s were my favorite decade. I was in high school, and my music ...
Different Treatment Strategies May Affect Sports Participation in Hemophilia Patients, Study Suggests
March 6, 2019March 6, 2019
Hemophilia patients who take intermediate‐dose preventive medication may experience more of an age-related decline ...
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 6, 2019March 6, 2019
As a New Mom, I Want Only the Best for My Daughter’s Health
March 4, 2019March 1, 2019
Hemophilia A Patients Report Benefits of Using FuseNGo Device, Study Shows
March 4, 2019March 4, 2019
Scientists Combine CRISPR-Cas9, Adenovirus Gene Delivery to Restore Function of FIX in Mice
March 1, 2019March 1, 2019

Why Am I So Angry?
February 28, 2019February 27, 2019
In past columns, I discussed my frustrations when anger rears its ugly head out ...
I Have a New Perspective, but I Still Remember the Past
February 27, 2019February 26, 2019
Discovery of Hemophilia A Subgroups May Help Predict FVIII Inhibitor Development
February 27, 2019February 27, 2019
WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 25, 2019February 25, 2019

Relapse Does Not Affect Survival in Patients with Acquired Hemophilia A, Study Suggests
February 25, 2019February 25, 2019
The relapse rate is higher in older patients with acquired hemophilia A (AHA) treated with ...
FDA Approves Esperoct as Prophylactic Treatment for Hemophilia A, Novo Nordisk Says
February 22, 2019February 22, 2019
Vienna to Host RARE2019 Meeting on Rare Diseases
February 22, 2019February 22, 2019

Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Removing Port Number 7
February 21, 2019February 20, 2019
This week, we will consult with the surgeon who will have placed and removed ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Self-care Isn’t Selfish
February 20, 2019February 18, 2019
When you become a parent, your world instantly changes. Your life focus becomes the ...
News

Spark Therapeutics Provides Update on SPK-8011, Other Potential Gene Therapies for Hemophilia
February 20, 2019February 20, 2019
SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe ...
Columns, HemoWife - a Column by Alliah Czarielle

At Times, Letting Go of Perfection Is the Perfect Decision
February 18, 2019February 15, 2019
All of the items I own are covered with breast milk; it’s on my ...
News

Hemlibra Now Available to Patients in Developing Countries via Aid Program
February 18, 2019February 18, 2019
Hemlibra (emicizumab), the sole approved prophylactic treatment for hemophilia A, is now available to patients in developing ...
News

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 15, 2019February 15, 2019
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities ...
News

Preliminary Data Further Show MarzAA’s Potential to Prevent Bleedings
February 15, 2019February 15, 2019
Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

We Will Continue to Rebuild Our Lives
February 14, 2019February 13, 2019
February 2014 proved to host the last bleed “MacDonald the Younger” faced thus far. ...
News

Single Dose of Gene Therapy AMT-061 Proves Effective in Hemophilia B
February 14, 2019February 14, 2019
A single administration of AMT-061, an investigational gene therapy for people with severe and ...
Columns, The 'I' in Hemophilia - a Column by Jared Formalejo

My First Steps into Parenthood
February 13, 2019February 12, 2019
As I write this, I’m bedridden from a bleed in my right hip. I’m ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

What Are the Things That Most Defines Us?
February 13, 2019February 12, 2019
The following is a group activity I like to do in some of the ...
Columns, HemoWife - a Column by Alliah Czarielle

To My Husband, the Father of My Daughter
February 11, 2019February 8, 2019
To the father of my daughter, Thank you for being present from the very ...
News

Higher Doses of Adynovate is Effective as Preventive Treatment for Hemophilia A, Trial Suggests
February 11, 2019February 11, 2019
Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

This Is Way Beyond My Understanding
February 7, 2019February 5, 2019
My youngest son, wife, and I just returned from an appointment with our hematologist ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

My Son Progressed from Severe Hemophilia to Mild, and I’ll Take It
February 6, 2019February 4, 2019
My mighty warrior Caeleb is about to start a new chapter in his life ...

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 ... Read more

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