Rapid genomic testing helps 1,100 families worldwide target treatment for rare childhood disease

Rare disease families find roadmap to drug development at bootcamps

Ultragenyx’s Rare Bootcamp gives families a roadmap to help drive research and develop new treatments for rare diseases.

Rapid genomic testing helps 1,100 families worldwide target treatment for rare childhood disease

An international partnership designed to improve equality in access to genomic medicine for a rare disease has now provided potentially life-saving genetic testing for over 1,100 families across the world.