Treatment Perspectives and Evolving Paradigms in Rett Syndrome

David Lieberman, MD, PhD, describes Rett syndrome, including the various stages, incidence, and potential causes of this disease.

An expert explains how Rett syndrome is diagnosed, how commonly it is misdiagnosed, disease progression, and which providers are best positioned to manage a Rett Syndrome patient.

David Lieberman, MD, PhD, reviews the economic burden and long-term complications of Rett syndrome as well as highlights the unmet needs for patients.

An expert discusses the current treatment landscape of Rett syndrome including the goals of treatment and implications for utilization management strategies with the disease.

An expert provides an overview of the RSBQ and CGI score measurement tools and other tools/methods of assessment used in clinical practice.

David Lieberman, MD, PhD, discusses the results of the phase 3 LAVENDER trial of trofinetide and the phase 3 AVATAR trial of blarcamesine and how patients and families can learn about eligible clinical trials for possible participation.

David Lieberman, MD, PhD, reviews the potential role and the goals of gene therapy in the treatment of Rett syndrome.

Paige Nues, from the International Rett Syndrome Foundation, provides insights on the diagnostic journey for patients with Rett syndrome.

Patient advocate Paige Nues elaborates on the patient journey associated with a Rett syndrome diagnosis.

Patients and families facing a Rett syndrome diagnosis experience an array of burdens.

Considerations for insurance preauthorization and reauthorization are explored by a key opinion leader.

An expert highlights unmet needs in the treatment of Rett syndrome.

A key opinion leader shares considerations for seeking eligibility and enrollment in clinical trials for patients with Rett syndrome.

Rett syndrome significantly impacts both patient and family’s quality of life.

Paige Nues of the International Rett Syndrome Foundation explores the broader effect on a patient’s family members and caregivers.

Paige Nues of the International Rett Syndrome Foundation discusses the complexity of navigating available resources and programs for patients with Rett syndrome, and the critical role of centers of excellence.

Paige Nues provides her closing thoughts regarding the treatment management of Rett syndrome.