SMA Gene Therapy Patients Require Ongoing Respiratory Care

Children who have received gene therapy for spinal muscular atrophy need monitoring for residual sleep and physical concerns.

Updated on Oct. 27, 2026 in Asthma

Gouache-painted illustration of a single sculpted lung form, representing the ongoing respiratory monitoring needs in pediatric spinal muscular atrophy care. — Pulmonologists are calling for structured respiratory monitoring for children with spinal muscular atrophy who have undergone gene therapy treatment. AI Illustration. Upload story photo >

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Pulmonologists are calling for structured follow-up care for children with spinal muscular atrophy who have received gene therapy. While treatments have led to significant motor gains, many patients still face residual health challenges.

Why it matters

Despite the success of gene therapy in motor development, residual complications such as sleep-disordered breathing, swallowing issues, and scoliosis can persist. This shift in care ensures clinicians monitor patients for these specific ongoing respiratory and physical needs.

Clinical observations presented at the CHEST 2026 Annual Meeting highlight ongoing monitoring needs in pediatric populations following gene therapy. While these treatments are approved for specific age brackets, the exact long-term residual disease burden remains under investigation.

The players

Moshe Prero

A pediatric pulmonologist affiliated with University Hospitals Rainbow Babies and Children's Hospital.

FDA

The federal agency responsible for regulating gene therapies and other medical products for pediatric health.

The details

Gene therapy for spinal muscular atrophy, such as onasemnogene abeparvovec-xioi, has significantly improved motor function in patients. However, experts note that even with these gains, patients may develop swallowing dysfunction or scoliosis, which impacts respiratory health. Clinicians are advised to use sleep studies to screen for sleep-disordered breathing in patients reporting symptoms like snoring or daytime behavioral changes, and they may require nocturnal continuous positive airway pressure.

May 2019: FDA approved onasemnogene abeparvovec-xioi for spinal muscular atrophy.
December 2023: FDA approved gene therapies for patients with sickle cell disease.
October 21, 2026: Moshe Prero presented findings at the CHEST 2026 Annual Meeting.

Health Landscape

The medical community is evolving its approach to gene therapy by focusing on long-term management beyond the initial treatment phase. This shift follows the CHEST 2026 Annual Meeting and reflects a growing recognition that motor gains do not eliminate the risk of chronic respiratory issues.

If your child has received gene therapy for spinal muscular atrophy, track any changes in their sleep patterns or breathing habits. It is worth discussing with your doctor whether a sleep study is appropriate if you notice snoring or daytime behavioral changes.

The takeaway

Gene therapy for spinal muscular atrophy requires ongoing monitoring to address potential residual respiratory and physical challenges. Families should keep a record of any new snoring or sleep disturbances to review with their child's specialist.