Tell us a little about any current/previous work with other rare diseases. For example, have you ever been a part of a team that has worked on gene therapy/other drug development initiatives before, or has brought a potential treatment to a clinical trial? What has been one of your most rewarding experiences?
I have travelled the road from basic research to clinical trial several times. I also developed a gene therapy drug to treat advanced angina, now in late-stage clinical testing. To see actual clinical benefit in patients is the reward we work towards.

Why did you choose to work with the INADcure Foundation?
INADcure is a very focused and well-managed foundation who have defined their goals and moved decisively to achieve them. They are a pleasure to work with.

What recent breakthroughs have gotten you most excited for the future of rare disease?
Although I am not sure where it is going, the new somatic gene editing methods are showing great potential for treating some genetic diseases. I hope to participate in programs to make that a reality.

What is your favorite part of the work you do?
The foundations which I work for and our academic collaborators are a community fully committed to the goal of developing treatment for genetic diseases. It deeply satisfying to be a part of that effort and to contribute as best I can.

What do you do to relax (or have fun)?
The endless distractions of the big city.

If you could have a lifetime supply of anything, what would it be?
Energy

Last (or Best) book read?
Let the Great World Spin

If you could have one superpower, what would it be?
Never sleeping but not being tired!