News

October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

News

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit ... Read more

October 26, 2018October 26, 2018

Bayer’s rFVIII Replacement Therapy Jivi Approved in Canada for Hemophilia A Patients 12 and Older

News

Hemophilia A patients age 12 and older who live in Canada now may receive prophylactic (preventive) treatment with Bayer’s antihemophilic factor Jivi. Health Canada approved Jivi as a treatment to prevent ... Read more

October 23, 2018October 24, 2018

AVV-Gene Therapy Successfully Given Again to Dogs in Model of Hemophilia A

News

Re-administering a viral vector-based gene therapy for hemophilia A was shown to be safe and to achieve long-term improvement in a dog model of the disease — even after ... Read more

October 18, 2018October 18, 2018

Students Chosen for “Hemophilia: The Musical” Production Announced by BioMarin and Believe Limited

News

BioMarin Pharmaceutical Inc. and Believe Limited have announced the students chosen to participate in the theatrical production of “Hemophilia: The Musical,” a program that aims to empower the bleeding community through performance ... Read more

October 18, 2018October 18, 2018

Octapharma USA Sponsors Retreat for Women With Bleeding Disorders and Screens ‘Bombardier Blood’

News

There are still too many women with bleeding disorders who, despite symptoms, are unaware of their disease. That circumstance will be a major topic Oct. 19-21 at The Fourth ... Read more

October 15, 2018October 15, 2018

Rate of Total Joint Replacement Similar in Hemophilia A and B, Study Reports

News

Total joint replacement (TJR) is more common in severe hemophilia patients requiring more frequent treatment, according to a population-based study in Taiwan. The findings also revealed that the incidence ... Read more

October 12, 2018October 12, 2018

Bypassing Agents Used for Hemophilia A May Also Benefit Children with Hemophilia B, Case Series Reports

News

Single or combination therapy with bypassing agents used for hemophilia A patients with inhibitors can be suitable treatment options for children with hemophilia B with factor IX inhibitors as well, ... Read more

October 10, 2018October 10, 2018

FDA Approves Hemlibra to Treat Hemophilia A Without Factor VIII Inhibitors

News

The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors. Hemlibra is now the ... Read more

October 9, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products ... Read more

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