Updates

September 25, 2020September 25, 2020
‘Advancements’ TV Show to Feature State-of-the-Art Research in Bleeding Disorders

An upcoming episode of the TV series “Advancements,” hosted by Ted Danson, will be dedicated to current and future research and therapeutics for inheritable ... Read more
September 24, 2020September 22, 2020
I Am Blessed With a Strong Community

When our youngest son, whom we affectionately call “MacDonald the Younger,” was diagnosed with hemophilia, my wife and I didn’t know how to respond. ... Read more
September 23, 2020September 21, 2020
My ‘Teapot’ Emergency Fund Gives Me Peace of Mind

Quarters. I ended up with a fistful of quarters the other day and had flashbacks to the early days of hemophilia. When my firstborn ... Read more
September 23, 2020September 23, 2020
GC Pharma, Atomwise Aim for Potential Oral Hemophilia Therapies

Atomwise, a company that specializes in using artificial intelligence (AI) to develop small molecule medicines, will partner with GC Pharma to discover new ways of ... Read more
September 22, 2020September 18, 2020
Finding Hope and Community Through the Hemophilia Forums

You are not alone. Coping with hemophilia as a patient, caretaker, or medical provider can seem overwhelming at times. When I am overwhelmed, have ... Read more

Columns, HemoWife - a Column by Alliah Czarielle

Sticking to Our Niche as a PwD-run Business
September 21, 2020September 17, 2020
When my husband, Jared, and I first started our online jewelry store, we knew ...
News

Mononine Soon Will No Longer Be Available to Treat Hemophilia B
September 21, 2020September 22, 2020
Mononine, a long-standing treatment to prevent and control bleeding in hemophilia B, will be discontinued ...
News

Roche Proposes Transfer Needle With Filter When Using Hemlibra
September 18, 2020September 18, 2020
Reports of translucent particles of being detected in Hemlibra (emicizumab) in 2019 do not ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Developing a Strong Work Ethic With Hemophilia
September 17, 2020September 15, 2020
From early on, we find success by developing a strong work ethic. Some of ...
News

Cancer Therapy Daratumumab Can Effectively Lower Inhibitors in Hemophilia A, Researchers Say
September 16, 2020September 16, 2020
Daratumumab — a cancer therapy marketed as Darzalex — can reduce levels of factor ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

How Would I Have Handled the Hemophilia Journey?
September 16, 2020September 15, 2020
Sometimes I am overwhelmed when I think about the hemophilia journey my sons are ...

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Discovering How Alone Time Can Help Me
September 14, 2020September 10, 2020
Lately, I’ve been finding myself in need of more “alone time.” Now that I ...
BIVV001 Safely Treats Severe Hemophilia A as Weekly Injection, Trial Reports
September 14, 2020September 14, 2020
A single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity ...
EMA Also Pushes Back Decision on Roctavian as Hemophilia A Gene Therapy
September 11, 2020September 11, 2020
Our Pets Teach Us the Darndest Things
September 10, 2020September 8, 2020
During Pain Awareness Month, It’s Important to Share Your Story
September 9, 2020September 8, 2020
Carton Label Mix-Up Causes Nationwide Recall of Tranexamic Acid Injection
September 9, 2020September 9, 2020

Combating 4 Common Myths About Hemophilia
September 8, 2020September 3, 2020
When I have the opportunity to gather with my brothers and sisters from the ...
Rare Case of Abnormal Bone Growth Successfully Treated With Surgery, Report Shows
September 4, 2020September 4, 2020
Focusing on Mental Health at This Year’s Hemophilia Federation Symposium
September 3, 2020September 1, 2020
Same But Different Photo Contest Celebrates People With Rare Diseases
September 3, 2020September 3, 2020

Learning Life Lessons Little by Little
September 2, 2020August 31, 2020
My 24-year-old son, Julian, lives out of state, where he attends college and works ...
Genentech Opens HemeWork to Help Patients Realize Life Goals
August 31, 2020September 1, 2020
Establishing a Routine for Our Toddler’s Stability
August 31, 2020August 27, 2020

News

NHF Bleeding Disorder Education Programs Receive $500K CDC Grant
August 28, 2020August 28, 2020
The National Hemophilia Foundation (NHF) has received a $500,000 grant from the Centers for ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

How to Avoid the Shame and Blame Game
August 27, 2020August 24, 2020
I will never forget the first time the Hemophilia Federation of America asked me ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Facing Our Fears and Discovering Freedom
August 26, 2020August 24, 2020
A new mom recently called me to share her concerns and to ask questions ...
News

RNA in Nanoparticles Leads to Efficient FVIII Production in Mice, Study Finds
August 26, 2020August 26, 2020
Tiny particles containing RNA led to successful delivery and production of factor VIII (FVIII) ...
News, syndicated

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
August 25, 2020August 25, 2020
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

Knowing When to Move On
August 25, 2020August 24, 2020
We’ve all had them: those amazing flings that morph into less-than-ideal relationships. During the ...
Columns, HemoWife - a Column by Alliah Czarielle

Unconditional Love Is Necessary for Marriage and Parenting
August 24, 2020August 20, 2020
I’ve been reading a lot of parenting books lately. Now that our baby girl, ...
News

Hemlibra Safely Treats Children With Severe Hemophilia A, Real-Life Study Finds
August 24, 2020August 24, 2020
Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and ...
News

‘Huge Step Back’ for Hemophilia Gene Therapy, Expert Says of FDA Delay on Roctavian
August 21, 2020August 24, 2020
The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

My Takeaways From This Year’s Bleeding Disorders Conference
August 20, 2020August 18, 2020
Despite the challenges associated with holding a large conference online, the National Hemophilia Foundation ...
News

FDA Delays Decision on Roctavian, Potential Hemophilia A Gene Therapy, for a Year or More
August 19, 2020August 21, 2020
The U.S. Food and Drug Administration (FDA) has pushed back by more than one ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Remembering and Honoring the Ones Who Made a Difference
August 19, 2020August 17, 2020
When I think back to the early days of raising my first of two ...
News

National Hemophilia Foundation Offering Grants to Bleeding Disorder Groups
August 19, 2020August 19, 2020
The National Hemophilia Foundation (NHF) has opened a grant program to support the local ...
News, syndicated

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
August 18, 2020August 18, 2020
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. ...
News

Rebinyn/Refixia Effective and Safe in Untreated Boys With Hemophilia B, Data Suggest
August 17, 2020August 18, 2020
Refixia or Rebinyn (nonacog beta pegol, or N9-GP), an approved replacement therapy for hemophilia B, ...

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

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