Our focus

We are focusing on the development of treatments for neuromuscular and pulmonary diseases that currently lack treatment options, such as Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF).

We have dedicated more than a decade to researching potential treatments in our ongoing mission to make effective medicines available for these often overlooked, life-altering diseases. We are passionate about improving patients’ quality of life and our vision is to be a leader in providing treatments for rare diseases in these areas with high unmet medical needs.

Meet our Board of Directors

Meet our Executive Committee

Meet our Extended Management Team