Peer Exchange

  

Spinal Muscular Atrophy (SMA): Physician and Patient Management


Philippa Cheetham, MBChB, MRCS, MD, FRCS, leads a discussion on an overview of spinal muscular atrophy, with special consideration for initial patient presentation, and routine screening.

The typical patient populations and physical consequences of spinal muscular atrophy.

Sebastian Mills discusses his early life with spinal muscular atrophy, including his initial symptoms and his first assessment with Crystal Proud, MD.

Key opinion leaders discuss the diagnostic workup of spinal muscular atrophy, including the importance of genealogical assessment and SMA categorization.

Sebastian Mills and his SMA specialist, Crystal Proud, MD, discuss the initial steps for treatment, and the impact it has had on his life.

Philippa Cheetham, MBChB, MRCS, MD, FRCS, leads a discussion on the possible drawbacks of spinal muscular atrophy treatment, including inaccessibility due to cost, regional access, and mode of delivery.

A discussion on how to treat children with spinal muscular atrophy, and the risks of spinal tap therapy.

A consideration of the possible systemic adverse effects from spinal muscular atrophy treatment, and dose modification due to patient response.

Key opinion leaders discuss life after beginning therapy and consider when to change therapy due to negative patient response.

A discussion on the importance of a multidisciplinary team when treating patients with SMA, and educational resources for community practitioners, patients, and caregivers.

Philippa Cheetham, MBChB, MRCS, MD, FRCS, leads a discussion on the long-term management of spinal muscular atrophy, and the importance of genetic testing for individuals with SMA who are planning a family.

A discussion on the importance of being treated at centers of excellence, and the future of oral medication in treating SMA.

The psychological impact spinal muscular atrophy can have on children and adults alike.

A discussion on the ENDEAR trial regimen and how an FDA approved treatment has impacted the management of spinal muscular atrophy.

Crystal Proud, MD, discusses ongoing clinical trials for spinal muscular atrophy, and provides advice for how to manage patient expectations.

Crystal Proud, MD, considers the possibility of incorporating neuromuscular treatments for spinal muscular atrophy, and the complications of receiving combination therapies.
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