Updates

March 25, 2019March 25, 2019
Population-Based Pharmacokinetic Dosing May Benefit Hemophilia A Patients, Study Reports

A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual ... Read more
March 22, 2019March 22, 2019
Healthcare Costs Higher with Bypassing Agents than Factor Replacement Therapy in Hemophilia A, Study Reports

Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study ... Read more
March 21, 2019March 19, 2019
Be Careful What You Say and Do!

My wife and I learned early to be careful when discussing the struggles we share in caregiving. Sometimes we get tired and want to ... Read more
March 20, 2019March 18, 2019
Bleeding Disorders Awareness Month: The Comfort of a Port

The unimaginable has happened. My youngest son, Caeleb, had his port removed. Of course, when someone with a bleeding disorder has a port, it’s ... Read more
March 20, 2019March 20, 2019
New Biomarker May Help Predict Response to Preventive Treatment with Factor VIII, Study Shows

New evidence suggests that a parameter called FVIII-EC50 has potential as a new way to predict the response of an individual with severe hemophilia ... Read more

News

Prophylaxis Partially Prevents Bleeding in Severe Hemophilia A Patients, Real‐world Study Finds
March 18, 2019March 18, 2019
Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and ...
News

Hemlibra Approved in Europe to Prevent Bleeds in Hemophilia A Patients Without Factor VIII Inhibitors
March 15, 2019March 15, 2019
People with severe hemophilia A living in Europe can now receive treatment with Hemlibra ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Ready, Set … Off to the Hospital?!
March 14, 2019March 13, 2019
So, we are in the car. Everyone is in their respective seats. We are ...
News

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 14, 2019March 14, 2019
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will ...
Columns, The 'I' in Hemophilia - a Column by Jared Formalejo

The Struggles of Dating with a Chronic Illness
March 13, 2019March 12, 2019
Looking at myself now, my younger self never would have expected me to be ...
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.” ...

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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 ...
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 ...
Erectile Dysfunction More Prevalent with Age in Men With Hemophilia, Study Says
March 11, 2019March 11, 2019
Advate is Safe, Effective Therapy for Hemophilia A, Japanese Study Finds
March 8, 2019March 8, 2019
The Unique MacDonald Family Vocabulary
March 7, 2019March 6, 2019
Bleeding Disorder Awareness Month: The Hemophilia Holocaust
March 6, 2019March 4, 2019

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
Scientists successfully combined the gene-editing tool CRISPR-Cas9 and a special type of virus to ...
Why Am I So Angry?
February 28, 2019February 27, 2019
I Have a New Perspective, but I Still Remember the Past
February 27, 2019February 26, 2019

News

Discovery of Hemophilia A Subgroups May Help Predict FVIII Inhibitor Development
February 27, 2019February 27, 2019
Researchers have identified four distinct subgroups of hemophilia A patients according to their profile ...
News

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 25, 2019February 25, 2019
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ...
News

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 ...
News

FDA Approves Esperoct as Prophylactic Treatment for Hemophilia A, Novo Nordisk Says
February 22, 2019February 22, 2019
Children and adults with hemophilia A in the United States may soon be treated with ...
News

Vienna to Host RARE2019 Meeting on Rare Diseases
February 22, 2019February 22, 2019
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city ...
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 ...

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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