Rare Disease Research and Integrated Systems: 1+1=3
Rare Disease Research and Integrated Systems: 1+1=3 Rare disease data can be hard to come by…Really hard when there are fewer subjects to study compared to those in the general population. By their very nature, these data involve the need to be collected from more sparsely located subjects and researchers, and more data sources. Even [...]
MOVING GENE THERAPY INTO HIGH GEAR – Innovations from Children's Hospital Boston
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes. International collaboration has been critical. Children’s Hospital Boston is one [...]
Clinical Trials for Rare Disease Patients – Legislation needs your support!
We wanted you to be aware of pending Federal legislation that may be significant for you and for our rare disease community. In June, legislation that enables individuals with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits was sponsored by Senators Ron Wyden (D-OR), Christopher Dodd (D-CT), Richard Durbin [...]