Updates

August 6, 2020August 3, 2020
National Hemophilia Conference Provides Hope for the Future

Saturday afternoon marked the opening of the virtual edition of the National Hemophilia Foundation’s Bleeding Disorders Conference. I immediately missed the in-person experience, meeting ... Read more
August 5, 2020August 3, 2020
Writing and Sharing Our Stories Can Help Us Heal

A couple friends came over for lunch last weekend, and one started talking to my youngest son, Caeleb, who is 14. Caeleb and I ... Read more
August 5, 2020August 5, 2020
FVIII Prophylaxis Early in Life Improves Joint Outcomes in Hemophilia A, Study Shows

Starting factor VIII (FVIII) preventive therapy before the age of 2.5 years improves joint outcomes in young adults with hemophilia A compared to starting ... Read more
August 3, 2020July 30, 2020
Weathering Our Storms As a Loving Family

Monsoon season has begun here in the Philippines. Every time I drive in downtown Manila, I’m reminded of a family trip last year to ... Read more
August 3, 2020August 3, 2020
FDA Clears CRYOcheck Lab Test of Factor VIII Activity in Hemophilia A

The U.S. Food and Drug Administration (FDA) has cleared for use CRYOcheck Chromogenic Factor VIII, Precision BioLogic‘s lab test to monitor factor VIII activity in the blood ... Read more

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

Our Virtual Connectedness
July 30, 2020July 28, 2020
As we continue to practice social distancing, I find it exceedingly difficult to stay ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

These Comments Make Me Uncomfortable as a Parent
July 29, 2020July 27, 2020
When people find out my sons have hemophilia, their reactions often make me uncomfortable. ...
News

Enzyre Secures €1.4M to Advance Portable Device to Monitor Coagulation
July 29, 2020July 29, 2020
Novalis Biotechnology is supporting a €1.4 million ($1.64 million) investment in Enzyre to finalize ...
Columns, HemoWife - a Column by Alliah Czarielle

When Hemophilia Isn’t the Worst Enemy
July 27, 2020July 23, 2020
My husband, Jared, has had hemophilia since he was born. He has known no ...
News

#ISTH2020 – Unmet Medical Need Found in Mild, Moderate Hemophilia A Care
July 27, 2020July 27, 2020
Adults with mild and moderate hemophilia A have a substantial number of bleeds per ...
News

#ISTH2020 – Gene Therapy BAY 2599023 Showing Steady FVIII Activity, Safety in Trial
July 24, 2020July 28, 2020
A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production ...

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Beyond Diagnosis, We Found Community
July 23, 2020July 20, 2020
I am the first person to insist that a diagnosis does not define my ...
#ISTH2020 – Eloctate Effective in Previously Untreated Hemophilia A Patients, Trial Shows
July 22, 2020July 27, 2020
Treatment with Eloctate (efmoroctocog alfa) was well-tolerated and effective at preventing and treating bleeding ...
Going Back to School With a 504 Plan on Standby
July 22, 2020July 20, 2020
Celebrating Our Personal Victories
July 21, 2020July 20, 2020
#ISTH2020 – BioMarin’s Gene Therapy Drives Sustained FVIII Production
July 20, 2020July 21, 2020
We Are Teaching Our Toddler to Be Independent
July 20, 2020July 16, 2020

#ISTH2020 – FLT180a Gene Therapy Shows Promise for Hemophilia B Patients in Phase 1/2 Trial
July 17, 2020July 17, 2020
A single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained ...
We Are the Experts of Our Own Stories
July 16, 2020July 15, 2020
#ISTH2020 – Hemlibra Continues to Show Favorable Safety Profile, Early Phase 3b Trial Data Show
July 15, 2020July 20, 2020
Finding Joy During the Worst of the Worst
July 15, 2020July 13, 2020

#ISTH2020 – Gene Therapy SPK-8011 Lowers Bleeding Rate in Hemophilia A for up to 3 Years, Trial Shows
July 13, 2020July 13, 2020
A single dose of the investigational gene therapy SPK-8011 is safe and leads to ...
Life’s Struggles Are Real, but So Is Personal Growth
July 13, 2020July 9, 2020
Freeline Raises $120M for Pivotal Trial of FLT180a, a Gene Therapy Candidate for Hemophilia B
July 10, 2020July 10, 2020

News, syndicated

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
July 9, 2020July 9, 2020
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

The Roles We Play as Caregivers
July 9, 2020July 6, 2020
Two weeks ago, my family moved back to New Mexico. Today, I find myself ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Being Grateful for the Boring and Mundane
July 8, 2020July 6, 2020
I am a sucker for routine. Spontaneity does not come easily to me because ...
News

Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy
July 8, 2020July 8, 2020
CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

How to Help Your Doctor Take Hemophilia Seriously
July 7, 2020July 10, 2020
Last week, I met a new local hematologist. He is not my main hemophilia ...
News

Idelvion Approved in Europe for 21-day Prophylactic Dosing in Adults With Hemophilia B
July 6, 2020July 6, 2020
The European Medicines Agency (EMA) has approved 21-day dosing intervals for CSL Behring‘s Idelvion ...
Columns, HemoWife - a Column by Alliah Czarielle

Let’s Avoid Playing the Blame Game
July 6, 2020July 2, 2020
Social media is one of my guilty pleasures. Whenever I find myself with free ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Accepting a Hemophilia Diagnosis Was a Question of Faith
July 2, 2020June 29, 2020
I entered my 3-month-old son’s room and stared at him as he peacefully slept ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

When Keeping Up Appearances Isn’t Good Enough Anymore
July 1, 2020June 29, 2020
Some moments in my life are ingrained in my soul, such as the births ...
News

Access to Elocta for Hemophilia A Expanding in UK, Sobi Says
July 1, 2020July 1, 2020
People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog ...
Columns, HemoWife - a Column by Alliah Czarielle

Putting Disability in the Entrepreneurial Spotlight
June 29, 2020June 25, 2020
Over the past few months, my husband and I have been working extra hard ...
News

Sobi, Sanofi Again Donate Clotting Factors to WFH Aid Program
June 29, 2020June 29, 2020
Sobi and Sanofi announced an additional donation of up to 500 million international units ...
News

CSL Behring Acquires Rights to Market AMT-061, Gene Therapy for Hemophilia B
June 26, 2020June 26, 2020
CSL Behring has acquired exclusive global rights to commercialize AMT-061, a potential gene therapy for hemophilia ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Managing Chaos as a Family
June 25, 2020June 23, 2020
If there is one word in the English language that drives me batty, it’s ...
News

Investigational Gene Therapy Results in Huge Drop in Bleeding Rates and Factor VIII Usage, Updated Data Show
June 24, 2020June 24, 2020
Valoctocogene roxaparvovec, BioMarin Pharmaceutical‘s investigational gene therapy, results in a more than 90% drop ...

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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