For Patients

Our Promise

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

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Our Research

At Edgewise, we are committed to finding safe and effective treatments for individuals suffering from a wide range of muscle injury diseases.

Edgewise is currently focusing on the dystrophinopathies, Duchenne and Becker muscular dystrophy, certain forms of Limb girdle muscular Dystrophy and other adult skeletal muscle diseases. 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 and in combination with all approved and experimental therapies for this disease.

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 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).

For more information about this trial, please contact,

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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.

Critical Path Institute

Resources for Patients

Duchenne and Becker specific organizations:

Neuromuscular Disease focused organizations:

Rare Disease focused organizations:

Edgewise presentations and events: