Risk-benefit profile of onasemnogene abeparvovec in older and heavier children with spinal muscular atrophy type 1.
Neuromuscul Disord
; 42: 22-26, 2024 Sep.
Article
en En
| MEDLINE
| ID: mdl-39111255
ABSTRACT
Spinal muscular atrophy (SMA) is an autosomal recessive disorder with progressive muscle atrophy and weakness, caused by biallelic mutations in the survival motor neuron 1 (SNM1) gene. Onasemnogene abeparvovec (OA) is an approved gene replacement therapy for patients with SMA. We report on two patients with SMA type 1, weighing 20 kg, previously treated with Nusinersen, who received OA infusion at 7 years of age. To our knowledge, these two patients are the heaviest treated in the real-world and we describe their different courses after gene therapy, including liver impairment requiring long-term steroid treatment and additional immunosuppression, with only transitory improvement in functional outcomes. Our cases illustrate how careful risk-benefit consideration is required in treating older and heavier SMA patients with OA, especially in view of the multiple treatment choices available for older patients with SMA.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Productos Biológicos
/
Terapia Genética
/
Atrofias Musculares Espinales de la Infancia
Límite:
Child
/
Female
/
Humans
/
Male
Idioma:
En
Revista:
Neuromuscul Disord
Asunto de la revista:
NEUROLOGIA
Año:
2024
Tipo del documento:
Article
Pais de publicación:
Reino Unido