Peer Exchange

  

Understanding Spinal Muscular Atrophy (SMA) and the Recent Advances in Management


A panel of experts begin their discussion by reviewing the etiology and epidemiology of spinal muscular atrophy (SMA).

Given the spectrum of severity in spinal muscular atrophy (SMA), key opinion leaders consider drivers of difference and their impact on care.

A comprehensive review of the signs, symptoms, and tests involved in making a differential diagnosis of spinal muscular atrophy (SMA).

Experts reflect on the availability and value of genetic testing as it relates to the differential diagnosis of spinal muscular atrophy (SMA).

In light of the value of early treatment for spinal muscular atrophy (SMA), panelists shed light on newborn screening and management.

The panel come together to elucidate the current standard-of-care approaches to treating spinal muscular atrophy (SMA).

Opening the discussion on multidisciplinary care, experts consider the value of pulmonology in managing spinal muscular atrophy (SMA).

Key opinion leaders share a technical discussion on nutrition as it relates to optimizing the management of spinal muscular atrophy (SMA).

Continuing their discussion on the multidisciplinary management of spinal muscular atrophy (SMA), panelists evaluate the role of bone health.

A review of the evolving role of palliative care as it relates to the management of certain patients with spinal muscular atrophy (SMA).

Experts shed light on the data behind the first FDA approval for spinal muscular atrophy (SMA), nusinersen, and consider what was learned through its approval.

Taking into account data from several clinical trials, the panel discusses oral therapy for spinal muscular atrophy (SMA) with risdiplam.

Moving into the setting of gene therapy, key opinion leaders consider the potential of AVXS-101 (onasemnogene abeparvovec) for spinal muscular atrophy (SMA).

The panel of experts take evolving pharmacologics into account as they review the best treatment strategies for spinal muscular atrophy (SMA).

Following their discussion on the management of spinal muscular atrophy (SMA), expert panelists close with parting advice for practicing clinicians.
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