News

February 15, 2019February 15, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

News

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run ... Read more

February 15, 2019February 15, 2019

Preliminary Data Further Show MarzAA’s Potential to Prevent Bleedings

News

Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) in patients with Hemophilia A or B with inhibitors continues to demonstrate the treatment’s potential ... Read more

February 14, 2019February 14, 2019

Single Dose of Gene Therapy AMT-061 Proves Effective in Hemophilia B

News

A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled ... Read more

February 11, 2019February 11, 2019

Higher Doses of Adynovate is Effective as Preventive Treatment for Hemophilia A, Trial Suggests

News

Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number of bleedings and enables more patients to stop having bleeds, according to the first results ... Read more

February 6, 2019February 6, 2019

First Patient Treated in Phase 3 Trial of AMT-061 in Hemophilia B

News

A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety ... Read more

February 4, 2019February 4, 2019

Research into Physiotherapy and Rehab for Hemophilia Patients Needed, Review Says

News

People with hemophilia may benefit from physiotherapy and rehabilitation programs intended to manage bleeding and joint disease (arthropathy), but more research is needed to better understand those programs — and ... Read more

February 1, 2019

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

News

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal ... Read more

February 1, 2019February 1, 2019

Study Finds No Differences in Oral Health Quality of Life in Children With Hemophilia

News

Despite having lower oral health scores, children with hemophilia showed no differences in oral health-related quality of life compared to children without the condition, a study found. The study, “Parent’s report ... Read more

January 30, 2019January 30, 2019

Case Reports Call for Need to Customize Management of AHA for Elderly

News

Clinical symptoms of acquired hemophilia A in elderly patients can vary widely, a series of six case reports reveals. Also, these patients should be managed individually in order to ... Read more

January 28, 2019January 28, 2019

Cell-based Gene Therapy May Offer Long-term Treatment for Hemophilia A, Mouse Study Suggests

News

Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a stable and long-term treatment for hemophilia A, ... Read more

Pin It on Pinterest