Patients & Families

Individuals living with rare muscle diseases and their families are at the core of our work.

As we advance our research and potential treatments, we seek to keep the needs, experiences and hopes of individuals with rare muscle disorders and their families at the center of our mission.

View our patient stories

Our Guiding Principles

  • Start with patients. End with patients

  • Work with uncompromising standards

  • Communicate honestly and openly

  • Meet the challenge with urgency

We seek to understand the challenges patients, and their caregivers and families face in navigating everyday life to help us develop meaningful treatments.

View our pipeline

Patient Advocacy at Edgewise wants to:

  • Provide forums to gather input from individuals living with the diseases we work in to make meaningful contributions to how our clinical trials are designed
  • Connect patients, clinicians, scientists, and regulators to find new and better treatments
  • Work with advocacy organizations to align on community needs and shared goals
  • Provide resources to help educate individuals and families


Community resources

At Edgewise we are dedicated to our guiding principles.

Please feel free to contact us with questions and ideas:


Abby Bronson

Head of Patient Advocacy & External Innovation

Clinical Trials

The clinical trial process is an important aspect of bringing a new therapy to patients. It tests whether the potential treatment is safe and effective for the intended population. Each trial is designed to answer a different question and inform the next trial.  To learn more about Edgewise clinical trials click the button below.

Edgewise clinical trials

Our Expanded Access Policy

Edgewise Therapeutics (Edgewise) is a clinical-stage company dedicated to developing innovative therapies for rare muscle disorders, including Duchenne and Becker muscular dystrophy. We are committed to conducting clinical trials to evaluate the safety and efficacy of investigational therapies to obtain the necessary marketing approvals that allow physicians to prescribe them so individuals may have broad access.

In general, new investigational therapies must be reviewed and approved by a regulatory authority, such as the U.S. Food and Drug Administration (FDA), before they become commercially available.

In some circumstances, a treating physician may make a request to provide an unapproved therapy to a qualifying individual, which must be approved by the FDA and supported by the pharmaceutical manufacturer. This process is called expanded access, sometimes known as compassionate use. Expanded access programs are not offered by every pharmaceutical manufacturer or for every investigational therapy. Because our investigational therapies are in early testing and much remains unknown about their safety and efficacy, Edgewise does not currently offer an expanded access program.

Clinical trials are designed to evaluate whether an investigational therapy is safe and effective for patients. Data generated by clinical trials are evaluated by regulatory authorities to determine whether the investigational therapy can be approved for broad use. Edgewise believes that participation in a clinical trial is the most appropriate way to access our investigational therapies. We believe this supports the goal of advancing most rapidly towards making our investigational therapies available to as many as possible.

We encourage patients and their families to speak with their physicians about possible clinical trial participation. Information about our investigational therapies and ongoing clinical trials can be found on and We reserve the right to review and amend this policy in the future, including based on information from ongoing and future clinical development.