Changing the Lives of Patients with

Myotubular and Centronuclear Myopathies (CNM)

International Women’s Day

In recognition of International Women’s Day, we’d like to introduce you to one of our inspiring team members, our Head of Clinical Development, Dominique Nijkamp

I have more than 20 years of experience working in biotechnology, with a focus on Clinical Development and Quality, where I have been dedicated to helping to bring important therapies to patients with limited or no treatment options. In my role at Dynacure, I design clinical trials to demonstrate if a potential treatment can safely and effectively address the needs of a patient community, and I work to ensure that such studies are aligned to the feedback and insight we receive from patients, physicians, and regulatory authorities.

I oversee the design and execution of the clinical studies for our lead program DYN-101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM).

Last year, we began to dose the first patients through our Unite-CNM clinical study – a multicenter, ascending dose study Phase 1/2 clinical trial to evaluate DYN101. The trial is expected to enroll approximately 18 patients aged 16 years or older with X-linked myotubular myopathy (XLCNM) or autosomal dominant CNM (ADCNM). I am excited that as an organization we have entered this phase of development, with the potential to help patients who are desperately in need of treatment options. I am also excited about the potential of this investigational therapy to make a difference, and to learn from each heroic patient’s experience.

Always seek deeper understanding before searching for solutions.

In my work this means looking for the drivers behind what people say or do, asking why a regulation was created, or what the patients really need. Only with this level of understanding you can be truly creative and find solutions that ensure progress in your clinical program despite the inevitable challenges.  Seeking to understand first, will give you the opportunity to influence with impact in any area of your life.

Never hesitate to share your ideas with others. You may bring that much needed different perspective. And if not, take pride in your courage to be visible and engaged!

I enjoy spending time with my dogs as they light up my day with their joy for the outdoors.