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Articles & Podcasts

N=1 Collaborative: FDA’s New Plausible Mechanism Pathway

Articles & Podcasts

How a tailored ALS drug could help broaden rare disease therapies

From roadmap to a sustainable end-to-end individualized therapy pathway

The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK

Personalized medicine for rare neurogenetic disorders: can we make it happen?

Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

Annemieke Aartsma-Rus, Rob W.J. Collin, Ype Elgersma, Marlen C. Lauffer, Willeke van Roon-Mom ( Therapeutic Advances in Rare Disease )...

Articles & Podcasts

Employers Don't Want to Pay for Life-Saving Cures for Rare Diseases

How to pay for individualized genetic medicines

A Multistakeholder Perspective on Advancing Individualized Therapeutics

Illustrated Examples

‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

Illustrated Examples

All of Us and the Promise of Precision Medicine:

Illustrated Examples

MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE

Drug Regulation in the Era of Individualized Therapies

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