Long-Term Follow Up of Patients Receiving Novel Gene Therapy for SMA Type ILong-Term Follow Up of Patients Receiving Novel Gene Therapy for SMA Type I https://pediatricsnationwide.org/wp-content/themes/corpus/images/empty/thumbnail.jpg 150 150 Abbie Roth Abbie Roth https://pediatricsnationwide.org/wp-content/uploads/2021/02/062019ds5821_abbie-profile-new.jpg
- April 19, 2019
- Abbie Roth
Spinal muscular atrophy type 1 (SMA1) is a rare neuromuscular disease in which 75 percent of affected children die or require permanent ventilation by 13.6 months. Researchers recently published the long-term outcomes of patients who received the investigational drug AVXS-101 – an adeno-associated virus serotype 9 mediated gene replacement therapy.
Twelve children aged 1 to 8 months were treated and followed for 24 months. Longer-term follow up results are included as available for motor milestones.
Notably, for motor milestones, the patient who was the oldest at treatment achieved fewer milestones than those treated at younger ages.
Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbours O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatric Pulmonology. 2019;54:179-185.
Image credits: Adobe Stock (header); Nationwide Children’s (infographic)
About the author
You might also like
When Every Week Matters: Advancing a Treatment to the ClinicWhen Every Week Matters: Advancing a Treatment to the Clinic https://pediatricsnationwide.org/wp-content/uploads/2021/10/Learning-in-Real-Time-e1633354760908-1024x491.jpg 1024 491 Natalie Wilson Natalie Wilson https://pediatricsnationwide.org/wp-content/uploads/2021/06/Natalieheadshot3-2.png
The Importance of Body Composition in Cystic FibrosisThe Importance of Body Composition in Cystic Fibrosis https://pediatricsnationwide.org/wp-content/themes/corpus/images/empty/thumbnail.jpg 150 150 Mary Bates, PhD Mary Bates, PhD https://secure.gravatar.com/avatar/c6233ca2b7754ab7c4c820e14eb518c8?s=96&d=mm&r=g
Novel Approaches to Gene Therapies for Patients With Rare Genetic DiseasesNovel Approaches to Gene Therapies for Patients With Rare Genetic Diseases https://pediatricsnationwide.org/wp-content/themes/corpus/images/empty/thumbnail.jpg 150 150 Jessica Nye, PhD Jessica Nye, PhD https://pediatricsnationwide.org/wp-content/uploads/2021/09/JNye_glasses.png