Hemophilia News Today

September 23, 2020September 23, 2020

GC Pharma, Atomwise Aim for Potential Oral Hemophilia Therapies

Atomwise, a company that specializes in using artificial intelligence (AI) to develop small molecule medicines, will partner with GC Pharma to discover new ways of treating people with hemophilia. The partnership ... Read more

September 21, 2020September 21, 2020

Mononine Soon Will No Longer Be Available to Treat Hemophilia B

Hemophilia News Today

Mononine, a long-standing treatment to prevent and control bleeding in hemophilia B, will be discontinued due to declining demand in recent years. CSL Behring, the therapy’s manufacturer, said the decision was ... Read more

September 18, 2020September 19, 2020

Roche Proposes Transfer Needle With Filter When Using Hemlibra

Hemophilia News Today

Reports of translucent particles of being detected in Hemlibra (emicizumab) in 2019 do not pose a safety risk or affect the therapy’s effectiveness and, according to Roche, health authorities have ... Read more

September 16, 2020September 17, 2020

Cancer Therapy Daratumumab Can Effectively Lower Inhibitors in Hemophilia A, Researchers Say

Hemophilia News Today

Daratumumab — a cancer therapy marketed as Darzalex — can reduce levels of factor VIII (FVIII) inhibitors in people with hemophilia type A, researchers say. Their findings were published in ... Read more

September 14, 2020September 14, 2020

BIVV001 Safely Treats Severe Hemophilia A as Weekly Injection, Trial Reports

Hemophilia News Today

A single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity over one month in men with severe hemophilia A, lasting longer in the body than the ... Read more

September 11, 2020September 11, 2020

EMA Also Pushes Back Decision on Roctavian as Hemophilia A Gene Therapy

Hemophilia News Today

The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults ... Read more

September 9, 2020September 9, 2020

Carton Label Mix-Up Causes Nationwide Recall of Tranexamic Acid Injection

Hemophilia News Today

The global pharmaceutical company Mylan has voluntarily issued a nationwide recall of four lots of tranexamic acid injection — a generic form of Cyklokapron — and of amiodarone HCl injection, ... Read more

September 4, 2020September 4, 2020

Rare Case of Abnormal Bone Growth Successfully Treated With Surgery, Report Shows

Hemophilia News Today

In rare instances, people with hemophilia can grow bones outside their skeletal tissue — with bone growth found in muscles and soft tissues — which significantly limit their range of ... Read more

September 3, 2020September 4, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

Hemophilia News Today

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A ... Read more

September 2, 2020September 2, 2020

Fixx Pharmaceuticals Seeks to Cure Hemophilia With Gene Editing

Hemophilia News Today

Fixx Pharmaceuticals has launched operations ultimately aiming to find a cure for hemophilia through gene editing. Fixx was founded by Jesse John Francis Clark, an entrepreneur born with severe hemophilia and ... Read more

August 31, 2020September 1, 2020

Genentech Opens HemeWork to Help Patients Realize Life Goals

COVID-19 News, Hemophilia News Today

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 America ... Read more

August 28, 2020August 29, 2020

NHF Bleeding Disorder Education Programs Receive $500K CDC Grant

Hemophilia News Today

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 five-year ... Read more

August 26, 2020August 26, 2020

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

Hemophilia News Today

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 — in ... Read more

August 24, 2020August 24, 2020

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

Hemophilia News Today

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 was ... Read more

August 21, 2020August 21, 2020

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

Hemophilia News Today

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 in ... Read more