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Lunch & Learn: Gene Therapy for SMA

Spinal Muscular Atrophy (SMA) is a rare genetic disease that affects the motor nerve cells in the spinal cord and is the leading genetic cause of infant mortality. During this education session, caregivers will learn from experienced clinicians who will share insights on gene therapy options for SMA as well as a parent's perspective on having a child receive approved treatments. Presentations will be followed by a Q&A for attendees. This event is cohosted by Fighting for Kaiden FoundationLaughing at My Nightmare, and ASGCT.


11 – 11:05 a.m. 
ASGCT Welcome – Ali Kujawski, MPH 
Laughing at My Nightmare – Laura Burcaw
Fighting for Kaiden – Kelly Eakin  

Understanding Gene Therapy and Treatment
11:05 – 11:20 a.m. 
Speaker: Megan Waldrop, MD, Nationwide Children’s Hospital and Ohio State University 

This talk will introduce FDA-approved treatment options: how they work, how they are given, and eligibility considerations. There will be a review of some of the active clinical trials to understand dual treatments and expanding indications. The different pathways to access these treatments will also be discussed, including the role of newborn screenings, accessing approved therapies, and participating in a clinical trial. 

Caregiver Considerations  
11:20 – 11:35 a.m. 
Speaker: Susan Iannaccone, MD, FAAN, UT Southwestern Medical Center 

This talk will address risks and benefits of gene therapy, along with current outcomes for asymptomatic versus symptomatic individuals. There will also be guidance on how to decide which option is best for your child.  

A Parent's Experience
11:35 – 11:45 a.m. 
Speaker: Brian Council

Brian will share his family’s experience from an SMA diagnosis to his daughter receiving all three of the approved treatments. He will discuss what led his family down each treatment path and what lessons they learned along the way.

Moderated Q&A
11:45 a.m. – 12:00 p.m.  

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Last Updated: 01/30/2024

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