August 16, 2017August 16, 2017
Back to School: Hemo-mom Ready to Educate, Advocate
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald
My youngest son, Caeleb, begins middle school this year. He is super-excited about starting the sixth grade, but I ... Read more
August 16, 2017
Patient Surveys Can Help Doctors Do a Better Job of Treating Hemophilia, Study Suggests
News
Information from patient surveys can help doctors do a better job of managing hemophilia and improving patients’ outcomes, ... Read more
August 14, 2017August 14, 2017
HIV Therapies May Also Prevent Genetic Mutations That Cause Hemophilia, Study Finds
News
Researchers at South Dakota State University (SDSU) studying mouse sperm found that drugs such as those used to treat ... Read more
August 9, 2017August 9, 2017
Bleeding Disorder Community: Your Stories Matter
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald
Two likes. No comments. Just. Two. Likes. A friend, who has a son with hemophilia, told me about ... Read more
August 9, 2017
Biomarin Plans to Test Two Doses of BMN 270 in Phase 3 Trials for Treatment of Hemophilia A
News
BioMarin Pharmaceutical will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 ... Read more
August 4, 2017
Hemophilia Management Program Aims to Standardize Treatment Practices
News
A new hemophilia management program was launched as part of a collaborative agreement between Magellan Rx Management and ... Read more
August 2, 2017August 1, 2017
PE and 504s: A New Year is Beginning
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald
When my oldest son, Julian, started elementary school in 2001, I was encouraged to proceed with a 504 plan ... Read more
July 31, 2017July 31, 2017
Hemophilia B Gene Therapy AMT-060 Moves to Large-scale Production, Says UniQure
News
Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat ... Read more
July 26, 2017July 26, 2017
EU Grants €5.6 Million to Consortium Developing Sernova’s Cell Pouch Therapy for Hemophilia A Patients
News
The European Commission will award the Brussels-based HemAcure Consortium €5.6 million in next-stage funding of a therapy called Cell Pouch — developed ... Read more
July 26, 2017August 1, 2017
Finding Joy and Giving Thanks
Columns, Hemophilia 24/7 - a Column by Cazandra Campos-MacDonald
When you live with a bleeding disorder, life can sometimes seem to be nothing but chaos. Just as one ... Read more
July 24, 2017July 24, 2017
Chugai Pharmaceutical Files New Drug Application for Hemophilia A Treatment Emicizumab
News
Chugai Pharmaceutical filed a New Drug Application for the use of emicizumab as a potential treatment to prevent or ... Read more
July 21, 2017July 25, 2017
Phase 1 Trial of Potential RNA Therapy Fitusiran Shows Promise for Hemophilia A and B
News
Once-monthly injection of the investigational RNA therapy Fitusiran reduced the levels of the protein antithrombin, a natural inhibitor of ... Read more

My youngest son, Caeleb, begins middle school this year. He is super-excited about starting the sixth grade, but I am not nearly as enthusiastic. He’s already thinking about new ... Read more

August 16, 2017

Patient Surveys Can Help Doctors Do a Better Job of Treating Hemophilia, Study Suggests

News

Information from patient surveys can help doctors do a better job of managing hemophilia and improving patients’ outcomes, an American study reports. The research, which appeared in the journal Patient ... Read more

August 14, 2017August 14, 2017

HIV Therapies May Also Prevent Genetic Mutations That Cause Hemophilia, Study Finds

News

Researchers at South Dakota State University (SDSU) studying mouse sperm found that drugs such as those used to treat HIV infection suppress L1s, or snippets of DNA that cause ... Read more

August 9, 2017August 9, 2017

Bleeding Disorder Community: Your Stories Matter

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

Two likes. No comments. Just. Two. Likes. A friend, who has a son with hemophilia, told me about her daughter’s new diagnosis unrelated to bleeding disorders. She was scared ... Read more

August 9, 2017

Biomarin Plans to Test Two Doses of BMN 270 in Phase 3 Trials for Treatment of Hemophilia A

News

BioMarin Pharmaceutical will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing ... Read more

August 4, 2017

Hemophilia Management Program Aims to Standardize Treatment Practices

News

A new hemophilia management program was launched as part of a collaborative agreement between Magellan Rx Management and Health New England to standardize treatment practices and optimize pharmacy and ... Read more

August 2, 2017August 1, 2017

PE and 504s: A New Year is Beginning

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

When my oldest son, Julian, started elementary school in 2001, I was encouraged to proceed with a 504 plan for his protection. Section 504 refers to the Rehabilitation Act ... Read more

July 31, 2017July 31, 2017

Hemophilia B Gene Therapy AMT-060 Moves to Large-scale Production, Says UniQure

News

Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia B. This new platform will allow the Dutch company to ... Read more

July 26, 2017July 26, 2017

EU Grants €5.6 Million to Consortium Developing Sernova’s Cell Pouch Therapy for Hemophilia A Patients

News

The European Commission will award the Brussels-based HemAcure Consortium €5.6 million in next-stage funding of a therapy called Cell Pouch — developed in collaboration with Canada’s Sernova — for hemophilia A patients. The therapy ... Read more

July 26, 2017August 1, 2017

Finding Joy and Giving Thanks

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

When you live with a bleeding disorder, life can sometimes seem to be nothing but chaos. Just as one bleed seems to heal, another one begins. You start to ... Read more

July 24, 2017July 24, 2017

Chugai Pharmaceutical Files New Drug Application for Hemophilia A Treatment Emicizumab

News

Chugai Pharmaceutical filed a New Drug Application for the use of emicizumab as a potential treatment to prevent or decrease the occurrence of bleeding episodes in patients with hemophilia ... Read more

July 21, 2017July 25, 2017

Phase 1 Trial of Potential RNA Therapy Fitusiran Shows Promise for Hemophilia A and B

News

Once-monthly injection of the investigational RNA therapy Fitusiran reduced the levels of the protein antithrombin, a natural inhibitor of several components of the clotting system, and restored the balance ... Read more

Back to School: Hemo-mom Ready to Educate, Advocate