Patients & Families

Our Promise

At Edgewise, we know that patients and their families are at the core of everything we do. As we develop our science and potential treatments, Edgewise’s promise to patients and their families is to:

  • put the best interest of patients first
  • work with uncompromising standards in all we do
  • listen to the communities that we work in and be transparent in our communications
  • recognize that for rare disease patients, every day is a day too late and work with a sense of urgency.

Want to get in touch? Please contact info@edgewisetx.com.

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 https://www.edgewisetx.com and https://clinicaltrials.gov. We reserve the right to review and amend this policy in the future, including based on information from ongoing and future clinical development.

Our Research

At Edgewise, we are leveraging our unique platform to find safe and effective treatments for patients suffering from a wide range of degenerative muscle disorders.

In skeletal muscle diseases, skeletal muscle activity leads to membrane stress and muscle fiber breakdown, which leads to disease progression. We are designing strategies to stabilize muscle function and reduce muscle breakdown by limiting the ability of muscle fibers to damage themselves with use. This will preserve muscle fibers and also lessen inflammation and fibrosis due to muscle fiber breakdown.

Edgewise is currently focused on Duchenne and Becker muscular dystrophy, and certain forms of Limb girdle muscular dystrophy. Learn more about these diseases »

In skeletal muscle diseases, skeletal muscle activity leads to membrane stress and muscle fiber breakdown, which leads to more disease progression. We are designing strategies to stabilize muscle function and reduce muscle breakdown by limiting the ability of muscle fibers to damage themselves with use. This will preserve muscle fibers and also lessen the downstream effects that muscle fiber breakdown causes, such as inflammation and fibrosis.

In Duchenne and Becker muscular dystrophy, our approaches do not rely on dystrophin replacement so will be applicable for patients with any dystrophin mutation in addition to other skeletal muscle diseases.

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. For more information about the clinical trial process:

Active Clinical Trials

A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults

EDG-5506, our lead product candidate for Duchenne and Becker muscular dystrophy (DMD and BMD), is advancing in clinical development. An initial clinical trial is being conducted in healthy volunteers. We also plan to pursue clinical trials in patients with BMD and DMD depending on the results of the clinical trials and after regulatory discussions with the FDA and EMA.

For more information about this trial, please contact, info@edgewisetx.com

Learn more on clinicaltrials.gov »

Community & Resources

Edgewise is proud to partner with the patient advocacy communities we work in. We work actively with our partners on initiatives that accelerate the development of meaningful treatments and impact the lives of patients. Click on a logo to learn more about our partners.

Treat-NMD
Critical Path Institute

Resources for Patients

Duchenne and Becker specific organizations:

Neuromuscular Disease focused organizations:

Rare Disease focused organizations:

Edgewise presentations and events: