I agree with your comment, I shouldn't have generalized all pharma. We also work with number of small pharma but the experience with big-pharma has been negative for rare-disease research and small-pharma are constrained in several ways.

I am not in U.S., but my govt. prefers to fund low hanging fruit w.r.t rare-disease i.e. those disease in which mechanistic details are clear and that has better probability of successful treatment; this ignores all those rare diseases which doesn't even have clear mechanistic details like GNE Myopathy.

After a long struggle, with help of corporate donors and a research institute we're now conducting a natural history study ourselves[1].

[1] https://www.gne-myopathy.org/