Working collaboratively with a multidisciplinary team of experts

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Filters for every decision we make

  • Singular Focus

    We are a life science company built solely to solve Duchenne muscular dystrophy. We are technology agnostic.

  • Patient Centric

    We start with Duchenne muscular dystrophy patients and their needs.

  • Holistic Approach

    We are addressing the full spectrum of disease manifestation, from the underlying genetic cause to symptom management.

  • Collaboration

    We are working together with all stakeholders -- industry, academia, government and community -- to identify and accelerate treatments.

  • Center of Excellence

    We are bringing together the best knowledge to address all aspects of the disease.

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