Progeria trial underscores challenge of rare diseases
The mother of a young boy with Progeria has paved the way to the world's first clinical trial into a potential treatment of the disease, which will require recruiting more than half of the 42 known patients in the world. The case underscores both the promise of new genetic research into potential treatments as well as the challenges researchers face in studying rare diseases. Progeria affects only one in every four million to eight million children, most of whom die from a stroke or heart attack by the age of 13.
- here's the article from The Wall Street Journal (sub. req.)
Related Articles:
FDA approves Shire's Elaprase for rare disorder. Report
NIH budgets $71M to study rare diseases. Report
FDA approves Novartis' Gleevec for five rare diseases. Report
Comments
Post new comment
Paid Research Reports
- RNA therapy: the next big thing after monoclonal antibodies?
- Biotech M&A Strategies: Deal assessments, trends and future prospects
- The Dermatology Market Outlook to 2013: Competitive landscape, pipeline analysis and growth opportunities
- Pipeline Insight: Cancer Overview - Breast, Gynecological, Genitourinary - Diverse drugs approaching the market for many tumor t
- Sales Force Effectiveness


