Updates

March 27, 2020March 27, 2020
Phase 3 Trial of AMT-061 Gene Therapy for Hemophilia B Exceeds Target Dosing

A Phase 3 clinical trial investigating gene therapy candidate AMT-061 (etranacogene dezaparvovec) has reached its target of dosing 50 participants with moderate or severe ... Read more
March 25, 2020March 23, 2020
I Understand What It’s Like to Fear the Unknown

Fear. Millions of people are living with insane amounts of fear due to the outbreak of the COVID-19 virus. This includes fear that the ... Read more
March 25, 2020March 25, 2020
FVIII Gene Therapy Directly into Joints May Best Protect Against Damage

Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy ... Read more
March 23, 2020March 20, 2020
The Coronavirus Pandemic Has Highlighted Societal Inequality

For the past three days, the capital of the Philippines has been on total lockdown due to the coronavirus outbreak. The country is now ... Read more
March 23, 2020March 23, 2020
Cell Therapy Seen to Ease Blood Loss in Hemophilia A Mouse Model

Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional ... Read more

News

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
March 20, 2020March 20, 2020
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate ...
Columns, Stories from the Road - a Column by Ann Kendall

Keep Your Swing in the Barrel
March 20, 2020March 19, 2020
I met my husband on the golf course. Now, one of my greatest joys ...
News

Sigilon Gets $80.3M to Support Trial of Hemophilia A Candidate SIG-001
March 20, 2020March 20, 2020
Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

As Parents, We Are in the Construction Business
March 19, 2020March 18, 2020
Raising children is like building a house. For those of us who manage chronic ...
Columns, The 'I' in Hemophilia - a Column by Jared Formalejo

When Hemophilia Meets Epilepsy
March 18, 2020March 17, 2020
It’s a struggle to balance life with two disorders. I need to take daily ...
News

Novo Nordisk Pauses 3 Clinical Trials of Concizumab Amid Safety Concerns
March 18, 2020March 27, 2020
Novo Nordisk has paused three clinical trials evaluating concizumab, its experimental treatment candidate for hemophilia ...

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How I React When Others Are Concerned About My Son’s Health
March 18, 2020March 17, 2020
It is hard for some of the people in my community to understand why ...
My Healthcare Providers Are Still Learning About Hemophilia
March 17, 2020March 16, 2020
Over the past few years, I have learned that bleeding disorders are complex, particularly ...
COVID-19 Has Me Stressed About Our Small Business
March 16, 2020March 12, 2020
New Gene Therapy Shows Promise in Hemophilia A Mouse Model, Study Shows
March 16, 2020March 16, 2020
Kovaltry Safe and Effective Prophylactic for Hemophilia A, Long-term Study Shows
March 13, 2020March 13, 2020
Music Is in the MacDonald Blood
March 12, 2020March 10, 2020

It’s Hard to See My Son Walk with a Cane
March 11, 2020March 9, 2020
My 14-year-old son Caeleb is having trouble walking. Some days are better than others, ...
Young Children, Parents Struggle Socially and Emotionally, Survey Finds
March 11, 2020March 11, 2020
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
March 9, 2020March 9, 2020
As We Get Older, Sometimes We Outgrow Friendships
March 9, 2020March 5, 2020

ClotChip, Portable Way of Analyzing Blood’s Ability to Clot, Named Breakthrough Device
March 9, 2020March 9, 2020
ClotChip, a portable device designed to quickly assess the ability of a person’s blood ...
In Potential $100M Deal, Medexus Acquires Ixinity for Hemophilia B
March 6, 2020March 6, 2020
Hemophilia Does Not Define My Sons
March 5, 2020March 3, 2020

News

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
March 4, 2020March 5, 2020
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

What It Means to Be Rare on Bleeding Disorders Awareness Month
March 4, 2020March 2, 2020
It seems as if there is a day for everything: there’s National Wear Red ...
News

Patients With Bleeding Disorders Given Global Voice in PPTA Awareness Campaign
March 4, 2020March 4, 2020
In support of Bleeding Disorders Awareness Month in March, the Plasma Protein Therapeutics Association (PPTA) is ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

It’s Not Easy Getting Appropriate Care
March 3, 2020February 28, 2020
Accessing quality healthcare can be a struggle. Women with hemophilia have added challenges that ...
News

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
March 2, 2020March 2, 2020
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review ...
Columns, HemoWife - a Column by Alliah Czarielle

I Find Healing in Music and a New Ukulele
March 2, 2020February 27, 2020
Recently, I’ve taken to the ukulele. I love its diminutive size, how its four ...
News

Low Doses of EHL-FIX Concentrates Can Prevent Bleeds in Severe Hemophilia B, Study Finds
March 2, 2020March 2, 2020
When given at low doses, extended half-life factor IX concentrates Alprolix (eftrenonacog alfa) and ...
Columns, Stories from the Road - a Column by Ann Kendall

Cookie Sheet, Muffin Tin, Cardboard: A Lesson for Rare Disease Week
February 28, 2020February 27, 2020
Science projects at home are often cause for consternation. We’ve all been there: Where ...
News

Obesity Likely Factor in Joint Bleeds and Pain in Hemophilia, Experts Say in US Survey
February 28, 2020February 28, 2020
Obesity increases the frequency of joint bleeds and degree of pain, and lowers overall ...
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald

Managing Multiple Hemophilia Diagnoses: How My Family Responded
February 27, 2020February 25, 2020
As I mentioned in an earlier column, the news that my firstborn son had ...
Columns, The 'I' in Hemophilia - a Column by Jared Formalejo

I’m Grateful for Childhood Friends Who Showed They Care
February 26, 2020February 24, 2020
Looking back at my younger self, I don’t think I fully appreciated the help ...
News

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
February 26, 2020February 26, 2020
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders ...
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald

Recognizing the Gift of Hemophilia
February 26, 2020February 24, 2020
Hemophilia has been a gift. Let me explain. I know some people will struggle ...
News

Hemophilia Patients Have Better Psychological Well-Being Than Other Adults, Study Reports
February 26, 2020February 26, 2020
People with hemophilia scored better on assessments of psychological well-being than healthy adults, a ...
Columns, The Forgotten Factor - a Column by Shellye Horowitz

My Self-esteem Was Shattered as a Child. Here’s How I Got It Back
February 25, 2020February 21, 2020
This week is National Eating Disorders Awareness Week. Eating disorders are tough to address, ...

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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