In this webinar, sponsored by IPG Health, we will explore the role that cell and gene therapies can play in treating rare diseases, the unique challenges faced by pharmaceutical companies, healthcare ...
High-quality evidence for the treatment of very rare diseases: IQWiG is involved in the EU project RealiseD Developing clinical trials to provide good evidence for very rare diseases, so that ...
News on rare diseases, including the development of new treatments, the challenges of diagnosis and treatment and the stories of patients and their families. The page features in-depth reporting ...
Rare diseases aren’t as rare as the appellation suggests. For instance, according to the US Government Accountability Office , 30 million Americans have a rare disease, a little less than 10% of ...