Rare Disease news

Rare Disease Video Recap—December 4, 2025

This biweekly Rare Disease Video Recap highlights regulatory designations,…


Cell_Gene_Therapy

Cell and Gene Therapy Today—November 27, 2025

This week's Cell and Gene therapy update covers major regulatory approvals,…


Rare disease drug development challenges

How can we address challenges in rare disease drug development?

It is estimated that rare diseases affect more than 400 million people…


Privacy Preference Center