News

August 31, 2020September 1, 2020

Genentech Opens HemeWork to Help Patients Realize Life Goals

News

Genentech has launched a program called HemeWork to support the professional development and career goals of people living with hemophilia. HemeWork was developed with input from the Hemophilia Federation of ... Read more

August 28, 2020August 28, 2020

NHF Bleeding Disorder Education Programs Receive $500K CDC Grant

News

The National Hemophilia Foundation (NHF) has received a $500,000 grant from the Centers for Disease Control and Prevention (CDC) to support education and outreach efforts in bleeding disorders. The ... Read more

August 26, 2020August 26, 2020

RNA in Nanoparticles Leads to Efficient FVIII Production in Mice, Study Finds

News

Tiny particles containing RNA led to successful delivery and production of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — ... Read more

August 25, 2020August 25, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News, syndicated

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

August 24, 2020August 24, 2020

Hemlibra Safely Treats Children With Severe Hemophilia A, Real-Life Study Finds

News

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness ... Read more

August 21, 2020August 24, 2020

‘Huge Step Back’ for Hemophilia Gene Therapy, Expert Says of FDA Delay on Roctavian

News

The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others ... Read more

August 19, 2020August 21, 2020

FDA Delays Decision on Roctavian, Potential Hemophilia A Gene Therapy, for a Year or More

News

The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In ... Read more

August 19, 2020August 19, 2020

National Hemophilia Foundation Offering Grants to Bleeding Disorder Groups

News

The National Hemophilia Foundation (NHF) has opened a grant program to support the local groups and chapters that work with bleeding disorder families, and have watched as their revenue ... Read more

August 18, 2020August 18, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. ... Read more

August 17, 2020August 18, 2020

Rebinyn/Refixia Effective and Safe in Untreated Boys With Hemophilia B, Data Suggest

News

Refixia or Rebinyn (nonacog beta pegol, or N9-GP), an approved replacement therapy for hemophilia B, is effective and has a good safety profile in previously untreated boys younger than 6 ... Read more

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