Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (<i>SMN1</i>) gene, which leads to a reduced level in the SMN protein within cells. Patients with SMA suffer from a loss of alpha motor neurons in the spinal cord l...
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MDPI AG
2023-06-01
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| Series: | Pharmaceutics |
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| Online Access: | https://www.mdpi.com/1999-4923/15/6/1764 |
| _version_ | 1797593071442984960 |
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| author | Charlotte A. René Robin J. Parks |
| author_facet | Charlotte A. René Robin J. Parks |
| author_sort | Charlotte A. René |
| collection | DOAJ |
| description | Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (<i>SMN1</i>) gene, which leads to a reduced level in the SMN protein within cells. Patients with SMA suffer from a loss of alpha motor neurons in the spinal cord leading to skeletal muscle atrophy in addition to deficits in other tissues and organs. Patients with severe forms of the disease require ventilator assistance and typically succumb to the disease due to respiratory failure. Onasemnogene abeparvovec is an adeno-associated virus (AAV)-based gene therapeutic that has been approved for infants and young children with SMA, and it is delivered through intravenous administration using a dose based on the weight of the patient. While excellent outcomes have been observed in treated patients, the greater viral dose necessary to treat older children and adults raises legitimate safety concerns. Recently, onasemnogene abeparvovec use was investigated in older children through a fixed dose and intrathecal administration, a route that provides a more direct delivery to affected cells in the spinal cord and central nervous system. The promising results observed in the STRONG trial may support approval of onasemnogene abeparvovec for a greater proportion of patients with SMA. |
| first_indexed | 2024-03-11T02:02:28Z |
| format | Article |
| id | doaj.art-b8ce72cff56f406696813e1c141d2ace |
| institution | Directory Open Access Journal |
| issn | 1999-4923 |
| language | English |
| last_indexed | 2024-03-11T02:02:28Z |
| publishDate | 2023-06-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceutics |
| spelling | doaj.art-b8ce72cff56f406696813e1c141d2ace2023-11-18T12:06:12ZengMDPI AGPharmaceutics1999-49232023-06-01156176410.3390/pharmaceutics15061764Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular AtrophyCharlotte A. René0Robin J. Parks1Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, ON K1H 8L6, CanadaRegenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, ON K1H 8L6, CanadaSpinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (<i>SMN1</i>) gene, which leads to a reduced level in the SMN protein within cells. Patients with SMA suffer from a loss of alpha motor neurons in the spinal cord leading to skeletal muscle atrophy in addition to deficits in other tissues and organs. Patients with severe forms of the disease require ventilator assistance and typically succumb to the disease due to respiratory failure. Onasemnogene abeparvovec is an adeno-associated virus (AAV)-based gene therapeutic that has been approved for infants and young children with SMA, and it is delivered through intravenous administration using a dose based on the weight of the patient. While excellent outcomes have been observed in treated patients, the greater viral dose necessary to treat older children and adults raises legitimate safety concerns. Recently, onasemnogene abeparvovec use was investigated in older children through a fixed dose and intrathecal administration, a route that provides a more direct delivery to affected cells in the spinal cord and central nervous system. The promising results observed in the STRONG trial may support approval of onasemnogene abeparvovec for a greater proportion of patients with SMA.https://www.mdpi.com/1999-4923/15/6/1764gene therapyonasemnogene abeparvovecneuromuscular diseasemotor neuron diseasespinal muscular atrophy |
| spellingShingle | Charlotte A. René Robin J. Parks Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy Pharmaceutics gene therapy onasemnogene abeparvovec neuromuscular disease motor neuron disease spinal muscular atrophy |
| title | Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy |
| title_full | Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy |
| title_fullStr | Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy |
| title_full_unstemmed | Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy |
| title_short | Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy |
| title_sort | expanding the availability of onasemnogene abeparvovec to older patients the evolving treatment landscape for spinal muscular atrophy |
| topic | gene therapy onasemnogene abeparvovec neuromuscular disease motor neuron disease spinal muscular atrophy |
| url | https://www.mdpi.com/1999-4923/15/6/1764 |
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