Hi all,
I know that I've seen several cases in the past where a disease has not been considered worthy of research--either due to its affecting only a very small population, or other utilitarian arguments. And in the cases I am thinking of, private citizens have formed coalitions to pool money and hire scientists to research the disease. For the life of me I can't recall the specific diseases, but this is along the same lines: The Wall Street Journal Reports On FDA Approval of Addi and Cassi’s Cyclodextrin Treatment | The Addi and Cassi Fund - Niemann Pick Type C So my question is HOW does one go about doing something like this? The article provided is not quite in line with the cases I am recalling, but states that:
No indication of how they went about that. This is a general question. I do not personally want to hire anyone at this moment. Even brainstorming would be great.Working with three other families whose children have NP-C disease, they hired a scientist who began writing a request to the FDA for the Hempel children to receive cyclodextrin infusions.![]()