Cell & Gene Therapy Insights: Curing ultra-rare diseases while ensuring access, equity and affordability

By definition, rare diseases are rare. Yet, while individual diseases may be rare and only affect a small population, the total number of people living with a rare disease is quite large. Surprisingly, if you amalgamate all the people living with the more than 7,000 known rare diseases, the population would be larger than the third most populous country in the world. And, one out of two patients diagnosed with rare disease is a child.

Read the full editorial article by R. Jude Samulski, Chairman, Chief Science Officer at Columbus Children’s Foundation & Laura Hameed, Executive Director at Columbus Children’s Foundation here.