May 17, 2018May 15, 2018
Finding Answers Together
Columns, In the Twinkling of an Eye - a Column by Joe MacDonald
Both of my sons have expressed frustration at having a bleeding disorder. After experiencing a problematic infusion, each has ... Read more
May 17, 2018May 17, 2018
‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35
News
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act ... Read more
May 17, 2018May 18, 2018
National Camp Exchange, Program for Hemophilia Camp Leaders, Now Taking Applications
News
The Arizona Hemophilia Association (AHA) is inviting camp professionals in the bleeding disorders and hemophilia communities to participate ... Read more
May 16, 2018
#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say
News
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live ... Read more
May 16, 2018May 15, 2018
Despite Hemophilia Joint Damage, New Treatment Opens Up Possibilities
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald
My mighty warrior Caeleb came home from school last week with a question. “Mom,” he asked, “my knee is really messed up, ... Read more
May 16, 2018May 16, 2018
Valoctocogene Roxaparvovec Study Doses 1st Hemophilia Patient with Pre-existing AAV5 Antibodies
News
A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with ... Read more
May 14, 2018May 14, 2018
Workshop on Factor VIII Inhibitors to Take Place This Week at NIH in Maryland
News
The National Heart, Lung and Blood Institute (NHLBI) is hosting a State of the Science Workshop on May 15-16, ... Read more
May 11, 2018May 11, 2018
IV Self-Infusion Training Significantly Reduced Infections After Joint Replacement Surgery
News
Teaching hemophilia patients who have undergone joint replacement surgery how to administer their intravenous (IV) self-infusions in sterility, ... Read more
May 9, 2018May 9, 2018
New Hemophilia Therapy Could Be a Game Changer
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald
With my oldest son, who is now 21, I never worried about limitations. He played soccer for a season, ... Read more
May 9, 2018May 9, 2018
On World Hemophilia Day, Bioverativ Shares Success Stories from the Developing World
News
In recognition of World Hemophilia Day 2018, the biopharmaceutical company Bioverativ is sharing the stories of people with hemophilia in ... Read more
May 7, 2018May 7, 2018
Type of Stem Cell in Blood Vessels Might Treat Bleeding Disorders, Mice Study Shows
News
A newly identified class of stem cells in blood vessels, called vascular endothelial stem cells, were able to repair injured vessels ... Read more
May 4, 2018May 4, 2018
Extended Half-Life Products Double Cost of Hemophilia Treatment, Prime Therapeutics Finds
News
The cost of hemophilia treatment doubled for patients who switched from standard half-life (SHL) to extended half-life (EHL) ... Read more

May 17, 2018May 15, 2018

Finding Answers Together

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

Both of my sons have expressed frustration at having a bleeding disorder. After experiencing a problematic infusion, each has cried out, “I hate having hemophilia!” While they are almost ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 17, 2018May 18, 2018

National Camp Exchange, Program for Hemophilia Camp Leaders, Now Taking Applications

News

The Arizona Hemophilia Association (AHA) is inviting camp professionals in the bleeding disorders and hemophilia communities to participate in the National Camp Exchange (NCE) program. Applications are open until ... Read more

May 16, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

May 16, 2018May 15, 2018

Despite Hemophilia Joint Damage, New Treatment Opens Up Possibilities

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

My mighty warrior Caeleb came home from school last week with a question. “Mom,” he asked, “my knee is really messed up, isn’t it?” I couldn’t see an injury, and so I ... Read more

May 16, 2018May 16, 2018

Valoctocogene Roxaparvovec Study Doses 1st Hemophilia Patient with Pre-existing AAV5 Antibodies

News

A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene ... Read more

May 14, 2018May 14, 2018

Workshop on Factor VIII Inhibitors to Take Place This Week at NIH in Maryland

News

The National Heart, Lung and Blood Institute (NHLBI) is hosting a State of the Science Workshop on May 15-16, 2018, in Bethesda, Maryland, to include input from the hemophilia ... Read more

May 11, 2018May 11, 2018

IV Self-Infusion Training Significantly Reduced Infections After Joint Replacement Surgery

News

Teaching hemophilia patients who have undergone joint replacement surgery how to administer their intravenous (IV) self-infusions in sterility, can decrease the rate of infections post-surgery significantly, a six-year education ... Read more

May 9, 2018May 9, 2018

New Hemophilia Therapy Could Be a Game Changer

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

With my oldest son, who is now 21, I never worried about limitations. He played soccer for a season, and that was the extent of his athletic pursuits. He ... Read more

May 9, 2018May 9, 2018

On World Hemophilia Day, Bioverativ Shares Success Stories from the Developing World

News

In recognition of World Hemophilia Day 2018, the biopharmaceutical company Bioverativ is sharing the stories of people with hemophilia in the developing world to highlight the life-changing potential of adequate treatment. ... Read more

May 7, 2018May 7, 2018

Type of Stem Cell in Blood Vessels Might Treat Bleeding Disorders, Mice Study Shows

News

A newly identified class of stem cells in blood vessels, called vascular endothelial stem cells, were able to repair injured vessels and ease bleeding symptoms in a mouse model of hemophilia, researchers ... Read more

May 4, 2018May 4, 2018

Extended Half-Life Products Double Cost of Hemophilia Treatment, Prime Therapeutics Finds

News

The cost of hemophilia treatment doubled for patients who switched from standard half-life (SHL) to extended half-life (EHL) products, according to a recent study by Prime Therapeutics. The findings ... Read more