The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) will hold a workshop for the public on rare disease advocacy. It will take place Monday, October 30, 2017.
The latest workshop expands on previously-held workshops.
As for the newly published strategic collaborative approach, the EMA and FDA discuss possible ways to enhance the efficiency of medicine development in Gaucher disease, a rare lysosomal storage disorder.
Rare disease research uncovers new mechanism underlying muscle development.
Learn more about Australia's reformed program for new orphan drug designations.
Gene therapy approval is imminent, with gene therapy coverage decisions coming soon after. With this approval, the initial indication and other factors will play a significant role.