News

LeaderMed, OPKO to Develop Treatments in Asia

Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental medications in China and other locations in Asia. One of the investigational medicines, Factor VIIa-CTP, is being developed as a potential treatment for hemophilia. “Through this joint venture, we…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Hemophilia Carriers Voice Need for Better Pregnancy, Childbirth Care

Women who are carriers for hemophilia — meaning they can pass the disease-causing mutation to their children — think a more individually tailored, information-rich healthcare approach could improve the experience of getting or being pregnant, a small study found. “Healthcare professionals could use these insights to adapt their consultations to…