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spinal muscular atrophy

Nationwide Children’s Hospital Completes One of the World’s First Prenatal Treatments for Spinal Muscular Atrophy
 Nationwide Children’s Hospital Completes One of the World’s First Prenatal Treatments for Spinal Muscular Atrophy 1024 600 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Baby Bryson laughing at a check up following treatment for SMA.

Investigational third-trimester, transplacental “bridging” therapy with risdiplam — followed by rapid postnatal treatment — aims to protect motor neurons during a critical window before and immediately after birth.  When Stacy Auker found out she was pregnant with her third child, she knew a diagnosis of spinal muscular atrophy (SMA) was a possibility.  “We have relatives who have been affected, so I…

Challenges in Clinical Decision-Making for Patients with Spinal Muscular Atrophy
 Challenges in Clinical Decision-Making for Patients with Spinal Muscular Atrophy 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

There are three approved genetic therapies for SMA, but little guidance in treatment selection. Spinal muscular atrophy (SMA) was once a leading cause of inherited infant death in the United States. Today, there are three genetic therapies approved by the Food and Drug Administration to treat SMA — but no clinical trials directly comparing treatment…