“We think we’ve got to bend the cost curve of these medicines. If we’re really going to have, in the years ahead, thousands of rare disease medicines, we have to be mindful of the cost to the system.”
John Crowley, CEO of Amicus Therapeutics Inc., which priced its newly FDA-approved Fabry disease drug Galafold (migalastat) at parity with Fabrazyme and pledged to never raise the U.S. price at a rate higher than the Consumer Price Index

“If a skeptic would say this is just a ploy [to advance the 2025 initiative], that strikes me as pretty harsh. But if a Pollyanna would say there’s no risk, I would say that’s a bit naïve.”
Philip Katz, partner at Hogan Lovells, on China’s Center for Drug Evaluation’s recent release of a list of 48 drugs approved elsewhere for rare diseases that could have an accelerated approval pathway in China

“The thrombin-delivery DNA nanorobot constitutes a major advance in the application of DNA nanotechnology for cancer therapy. In a melanoma mouse model, the nanorobot not only affected the primary tumor but also prevented the formation of metastasis, showing promising therapeutic potential.”
Hao Yan, director of the ASU Biodesign Institute’s Center for Molecular Design and Biomimetics and the Milton Glick Professor in the School of Molecular Sciences on the potential of nanotech-based oncology treatments

“I wish someone had warned me about the burden and stress of being a solo founder. It makes more sense at least to have a co-founder because then you have two parents for the company, which you think of as your child. There’s just no way around it: the company needs your attention all the time.”
Ethan Perlstein, founder and CEO of Perlara