Rare Diseases, Diagnosis, Therapies, and Hope

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Rare Diseases, Diagnosis, Therapies, and Hope
 
 

This is a free e-book on rare diseases written in a way that is easily accessible for all audiences.

The book includes:

  • Ten case studies about breakthrough discoveries for rare disease patients.

  • Many interviews with top doctors, researchers, and genomics experts.

  • A Rare Disease Parent’s Toolkit with recommendations from parents and others.

  • Demystifying Genomics, which explains in simple terms the latest developments in rare disease treatment and research, including information on how the sequencing process works.