spinal muscular atrophy
Rare disease · 2017–2025
12decisions
3medicines
10carried consumer input
1,363individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| risdiplam Treatment of spinal muscular atrophy (SMA) in adults and children aged 2 years or older weighing 20 kg or more. The tablet formulation is being listed as an… | Nov 2025 | Recommended with restriction | 1 individuals |
| risdiplam Treatment of pre-symptomatic patients with confirmed genetic diagnosis of spinal muscular atrophy (SMA) who have an SMN2 gene copy number of three. | Jul 2024 | Recommended with restriction | 1 individuals |
| onasemnogene abeparvovec Pre-symptomatic treatment of babies with spinal muscular atrophy (SMA) and 3 copies of the Survival Motor Neuron 2 (SMN2) gene. | Jul 2023 | Recommended | input received Spinal Muscular Atrophy Australia · Sydney Children Hospital Network |
| risdiplam Treatment of 5q spinal muscular atrophy (SMA) in three populations: (i) adults aged ≥19 years with symptom onset prior to age 19 and no prior disease-modifying… | Mar 2023 | Recommended with restriction | 22 individuals |
| onasemnogene abeparvovec Treatment of pre-symptomatic spinal muscular atrophy (SMA) patients with bi-allelic mutations in the SMN1 gene and 3 copies of the SMN2 gene, aged less than 9… | Nov 2022 | Not recommended | 22 individuals Spinal Muscular Atrophy Australia · National Network · Paediatric Neuromuscular Clinical Research Network |
| risdiplam Treatment of spinal muscular atrophy (SMA) Types 1, 2, 3a (in patients ≤18 years), Type 3b (in patients ≤18 years), and Types 1, 2, 3 (in patients >18 years). | Mar 2021 | Recommended with restriction | 219 individuals |
| nusinersen Treatment of pre-symptomatic, infantile-onset and childhood-onset spinal muscular atrophy (SMA) in patients with genetically confirmed SMN1 deletion or… | Jul 2020 | Recommended with restriction | 11 individuals Spinal Muscular Atrophy Australia · Rare Voices Australia |
| nusinersen Treatment of spinal muscular atrophy (SMA) in patients with symptom onset prior to 19 years of age, including Types I, II, IIIa, and IIIb SMA. | Nov 2020 | Not recommended | unclear |
| nusinersen Pre-symptomatic initiation of treatment of patients with Spinal Muscular Atrophy (SMA) who have SMN1 deletion or mutation with up to 3 copies of the SMN2 gene… | Jul 2019 | Not recommended | input received Spinal Muscular Atrophy Australia · Rare Voices Australia |
| nusinersen Pre-symptomatic initiation of treatment of patients with Spinal Muscular Atrophy (SMA) who have up to 3 copies of the survival-of-motor-neuron 2 (SMN2) gene… | Jul 2019 | Deferred | input received Spinal Muscular Atrophy Australia · Rare Voices Australia |
| nusinersen Treatment of paediatric patients with infantile-onset or childhood onset Spinal Muscular Atrophy (SMA) with onset of symptoms prior to 3 years of age. | Jul 2018 | Recommended with restriction | no section in document |
| nusinersen Treatment of infantile-onset (Type I) and childhood-onset (Types II and III) spinal muscular atrophy (SMA). | Nov 2017 | Not recommended | 1,087 individuals SMA Australia · Muscular Dystrophy Association |
Who spoke
Spinal Muscular Atrophy Australia · Rare Voices Australia · SMA Australia · Muscular Dystrophy Association · National Network · Paediatric Neuromuscular Clinical Research Network · Sydney Children Hospital Network
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 10 of 12 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.