Updates

May 29, 2020May 29, 2020
Infection by New Coronavirus Can Induce AHA, Case Report Says

Infection by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the coronavirus causing COVID-19, can induce the onset of acquired hemophilia A (AHA), a ... Read more
May 28, 2020May 26, 2020
And Then Came Aunt Jessie

Heroes come in all shapes and sizes. Sometimes, it is challenging to recognize the people in our lives who overcame overwhelming odds to remind ... Read more
May 28, 2020May 28, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 27, 2020May 27, 2020
FDA Agrees to Phase 1 Trial of ET3, Gene Therapy for Hemophilia A

The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of ET3, an investigational gene therapy for ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more

News

Rebinyn May Interfere With Tests for COVID-19-related Coagulation Problems, Novo Nordisk Says
May 22, 2020May 22, 2020
Novo Nordisk has issued a notice warning healthcare professionals that the hemophilia B treatment ...
cystic fibrosis, News, syndicated

Video Games Connect Chronically Ill Children Isolated at Home, Hospital
May 21, 2020May 21, 2020
Dara Riva always had a rule that her 10-year-old son could play video games ...
News

Long-term Rebinyn Can Safely Prevent Bleeds in Children With Severe Hemophilia B, Trial Shows
May 20, 2020May 20, 2020
Long-term treatment with Rebinyn (nonacog beta pegol, or N9-GP) is safe and effective at ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Being Open About Mental Health and Chronic Illness
May 20, 2020May 18, 2020
Editor’s note: Please be advised that the topic of suicide is addressed in this ...
News, syndicated

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
May 20, 2020
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

How I Mastered the Self-infusion of Factor Products
May 19, 2020May 18, 2020
When I was a young girl, I sat next to my father while he ...

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Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
May 19, 2020May 19, 2020
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first ...
My Mother’s Day Wish Is to Keep My Identity
May 18, 2020May 15, 2020
I celebrated Mother’s Day a week ago at home with my family. We held ...
3 Hemophilia B Patients Report AMT-060 Gene Therapy Gave Them Normalcy
May 18, 2020May 18, 2020
Hemophilia A Gene Therapy, AMT-180, Continues to Show Activity, Safety in Animals
May 15, 2020May 15, 2020
BioMarin’s Gene Therapy for Hemophilia A Remains on Track for US Approval
May 13, 2020May 13, 2020
What I Remember as the Mother of Newly Diagnosed Children
May 13, 2020May 11, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
May 12, 2020May 12, 2020
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better ...
4 Tips for Dealing with Caregiver Fatigue
May 11, 2020May 7, 2020
Risk of Bleeds in Hemophilia A Influenced by Treatment History, Other Factors, Study Suggests
May 11, 2020May 11, 2020
Generic Version of DDAVP to Treat Mild Hemophilia A Launches in US
May 8, 2020May 8, 2020

Surveys on the Sidewalk
May 8, 2020May 7, 2020
I first noticed the survey as I headed down the street while the sun ...
Life May Be Difficult, but We’ve Got This
May 7, 2020May 6, 2020
Letting My Child Grow Up While Remaining His Advocate
May 6, 2020May 4, 2020

News

EU Patients Satisfied with Treatment and Physically Active, Survey Finds
May 6, 2020May 6, 2020
Europeans with severe hemophilia are largely satisfied with available treatments and able to maintain ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

Protecting Hemophiliacs from the Unknowns of COVID-19
May 5, 2020May 1, 2020
Our hemophilia community has been here before. A novel virus — previously, it was ...
Columns, HemoWife - a Column by Alliah Czarielle

What Happens When the Pandemic Ends?
May 4, 2020April 30, 2020
What’s new, month of May? Why hello there, extended coronavirus lockdown! Our community ...
News

Prophylaxis Better Than On-demand Therapy for Reducing Bleeds in Hemophilia A, Study Reports
May 4, 2020May 4, 2020
Switching from on-demand to preventive treatment — or prophylaxis — significantly reduces bleeding in ...
News

Catalyst Biosciences Awarded EU Patent for Experimental Hemophilia B Therapies
May 1, 2020May 1, 2020
Catalyst Biosciences has received a European Union patent for its investigational hemophilia B treatments, ...
syndicated

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts
April 30, 2020April 30, 2020
As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise ...
Announcements

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak
April 29, 2020April 29, 2020
FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Remembering Our Other Family on the Front Lines
April 29, 2020April 30, 2020
Losing family members is hard. Long-lived lives that come to an end, distance that ...
News

Awareness Key to Proper Treatment Adherence in Hemophilia, Study Says
April 29, 2020April 29, 2020
Greater awareness of hemophilia and its symptoms is associated with better adherence to treatment by ...
News, syndicated

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
April 28, 2020April 28, 2020
While there are few silver linings to the cloud created by COVID-19, the pandemic ...
News

2 Proteins May Help Predict Acute Joint Bleeding in Severe Hemophilia A, Study Suggests
April 27, 2020April 27, 2020
Blood levels of two proteins — C-reactive protein (CRP) and vascular endothelial growth factor ...
Columns, HemoWife - a Column by Alliah Czarielle

Why We’re Planning to Get a Dog
April 27, 2020April 23, 2020
My husband, Jared, and I recently talked about getting a dog. I’m excited and ...
News

Hemlibra Can Interfere with Tests of COVID-19-associated Coagulation Problems, Genentech Says
April 24, 2020April 30, 2020
Genentech has issued a notice reminding healthcare professionals that its hemophilia A treatment Hemlibra ...
News, syndicated

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community
April 23, 2020April 30, 2020
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Giving Thanks for Peace While in Self-quarantine
April 22, 2020April 30, 2020
If I had to define my self-quarantine in one word, it would be peace. ...

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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