Case-Based Insights: Expert Perspectives on the Treatment of Adult-Onset SMA

Arun S. Varadhachary, MD, PhD provides an overview of the case of a 28-year-old male with spinal muscular atrophy, describes the 4 main phenotypes of SMA, and considers how average age of onset impacts patient prognosis.

An expert neurologist, Arun S. Varadhachary, MD, PhD considers the diagnostic process for SMA and emphasizes the importance of early diagnosis to optimize care.

James Wymer, MD, provides a case overview of a 38-year-old female diagnosed with spinal muscular atrophy.

An overview of the diagnosis of spinal muscular atrophy, including prevalence, age of onset, and the role of genetic testing.

A key opinion leader in neurology highlights the value of a multidisciplinary approach to care for patients with spinal muscular atrophy in order to maximize a patient’s function through physical and medical therapy.

A specialist in the care of patients with spinal muscular atrophy reviews the CHERISH trial of nusinersen and examines the drug’s safety and efficacy profiles.

A discussion of the multidisciplinary treatment approach in spinal muscular atrophy.

An insight into the goals of therapy and treatment selection between oral and intrathecal medications for adult-onset spinal muscular atrophy.

Arun S. Varadhachary, MD, PhD shares insights into clinicians’ and patients’ experiences with medical therapy, comments on unmet needs for adult patients, and speculates about the future of SMA care.

An expert neurologist emphasizes the importance of medication compliance and the value of multidisciplinary management in meeting patient goals of therapy for spinal muscular atrophy.

James Wymer, MD discusses his firsthand experience with risdiplam and identifies a key unmet need in the treatment of spinal muscular atrophy.