Updates

December 2, 2020December 2, 2020
Gene Therapy AMT-060 for Hemophilia B Prevents Bleeds Up to 5 Years, Ongoing Trial Shows

AMT-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and the use of clotting factor IX ... Read more
December 2, 2020November 30, 2020
December Is the Time to Take Inventory

Many people see spring as the season for cleaning. Winter months call for homes to be closed tightly to keep the heat in and ... Read more
December 1, 2020December 1, 2020
NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the ... Read more
November 30, 2020November 30, 2020
Most Hemophilia Patients Show Positive Attitude for Gene Therapy in Study

Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a ... Read more
November 26, 2020November 26, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their ... Read more

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

Today I Choose Thankfulness
November 25, 2020November 23, 2020
People are suffering. The country is divided. States are imposing limits on items that ...
News

First Patient Dosed in Phase 3 Trial Testing Marstacimab, Pfizer’s TFPI Therapy
November 25, 2020November 25, 2020
The first participant was dosed with the investigational therapy marstacimab in a Phase 3 ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

We Need a Standard of Care in Women’s Hemophilia Treatment
November 24, 2020November 20, 2020
I visited a hemophilia treatment center for the first time in the 1980s, to ...
Columns, HemoWife - a Column by Alliah Czarielle

Living One Day at a Time
November 23, 2020November 19, 2020
Lately I’ve been feeling stuck. My life seems to have taken on the appearance ...
News

Insurance Firm BCS Partners With NHF to Improve Healthcare Quality, Cost
November 23, 2020November 23, 2020
The healthcare insurance company BCS Financial has partnered with the National Hemophilia Foundation (NHF) ...
Columns, Stories from the Road - a Column by Ann Kendall

‘Tis the Season for Conquering Soup
November 20, 2020November 19, 2020
Long a food literature devotee, I continually marvel at the seeming effortlessness with which ...

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AMT-061 Increases FIX Activity, Reduces Bleeding in Phase 3 Trial, Data Show
November 20, 2020November 20, 2020
AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased ...
Maintaining Gratitude Through a Spirit of Thanks-living
November 19, 2020November 17, 2020
Let’s face it. This year contains one struggle after another. We continue to ask ...
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
November 19, 2020November 19, 2020
We Are Designed for Turmoil
November 18, 2020November 16, 2020
Enrollment Underway in Phase 4 Trial of Ixinity in Hemophilia B Patients Under Age 12
November 18, 2020November 18, 2020
When Broken Souls Heal Together
November 16, 2020November 12, 2020

DASH Diet Found to Yield Health Benefits in Adolescents With Hemophilia
November 16, 2020November 16, 2020
The Dietary Approach to Stop Hypertension (DASH) diet yields beneficial health effects, reducing blood ...
Sanofi Genzyme Places Trials for Fitusiran on Hold
November 13, 2020November 13, 2020
Life Lessons in the MacDonald Home
November 12, 2020November 10, 2020
Looking Back Is Still Painful
November 11, 2020November 9, 2020

Manual Therapy With Passive Stretching Helps Hemophilia Patients’ Joints, Study Finds
November 11, 2020November 11, 2020
Manual therapy, in combination with passive stretching, can reduce the frequency of joint bleeds ...
Keeping Moms and Babies Safe During Pregnancy and Birth
November 10, 2020November 9, 2020
My Cosplay Hobby Is a Good Way to Relieve Stress
November 9, 2020November 5, 2020

News

Scientists Identify New Genetic Mutations Underlying Hemophilia A
November 9, 2020November 9, 2020
Using a combination of genetic and functional approaches, scientists have identified new mutations in ...
News

Hemlibra Safely Prevent Bleeds in Hemophilia A Patients Over Long Term, Trial Data Show
November 6, 2020November 6, 2020
Hemlibra (emicizumab) continues to safely and effectively prevent bleedings in children and adults with hemophilia ...
Columns, Stories from the Road - a Column by Ann Kendall

The Leaves Among Us
November 6, 2020November 4, 2020
Fall has set in, and the leaves around us provide a colorful daily show ...
News, syndicated

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
November 5, 2020November 6, 2020
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Encountering Difficult People
November 5, 2020November 3, 2020
One day, my youngest son, whom we affectionately call “MacDonald the Younger,” and I ...
News

Hemophilia Treatments Should Be Open to All, Regardless of Cost, ICER Told
November 4, 2020November 4, 2020
The full range of hemophilia treatments should be made available to patients without consideration of cost ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

The Struggle to Have It All
November 4, 2020November 3, 2020
There are some very fortunate people who do not experience illness. They often come ...
Columns, HemoWife - a Column by Alliah Czarielle

The ‘Crab Mentality’ Makes the Struggle for Equality Difficult
November 2, 2020October 29, 2020
I am a staunch advocate of equality. From a very young age, I was ...
News

Adynovate Prophylaxis Continues to Show Promise in Severe Hemophilia A, Trials’ Data Show
November 2, 2020November 2, 2020
Prophylactic (preventive) treatment with Adynovate safely and effectively lowers the number of bleedings, including those ...
News

Acupuncture Might Work Better Than TENS to Reduce Chronic Joint Pain
October 30, 2020October 30, 2020
Acupuncture was found to be more effective for reducing chronic joint pain in hemophilia ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Choosing Which Factor Products to Use Was a Community Effort
October 29, 2020October 27, 2020
Early in the life of our son “MacDonald the Elder,” we came face to ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

There’s No Such Thing as a Silly Question
October 28, 2020October 26, 2020
Rarely am I the first to raise my hand in a class or give ...
News

Patients Report Mostly High Satisfaction With US Hemophilia Treatment Centers
October 28, 2020October 28, 2020
People living with bleeding disorders such as hemophilia and von Willebrand disease report consistently ...
News, syndicated

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
October 27, 2020October 28, 2020
A photo of a bespectacled young boy, his red baseball cap slightly askew as ...
Columns, HemoWife - a Column by Alliah Czarielle

My Husband and I Are Equal Partners
October 26, 2020October 22, 2020
In our marriage, my husband, Jared, and I are equals. He may have chronic ...

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 ... Read more