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MeteorMystic
Updated:10/05/2024
Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by weakness and wasting in muscles. Recent advancements in treatment offer hope for patients.
Treatment Options for SMA
- Nusinersen (Spinraza): An intrathecal injection that modifies SMN2 gene splicing to produce more SMN protein.
- Onasemnogene abeparvovec (Zolgensma): A gene therapy that delivers a functioning copy of the SMN1 gene.
- Risdiplam (Evrysdi): An oral medication that increases SMN protein levels by targeting the SMN2 gene.
Effectiveness of Treatments
| Treatment | Age Group | Effectiveness Rate | Notable Outcomes |
|---|---|---|---|
| Nusinersen | All ages | 40-60% | Improved motor function in infants and toddlers |
| Onasemnogene abeparvovec | < 2 years | 80-100% | Motor milestones achieved in most treated patients |
| Risdiplam | All ages | 60-70% | Improved survival and motor function |
Comparative Analysis
| Treatment | Administration Method | Frequency |
|---|---|---|
| Nusinersen | Intrathecal injection | Loading doses and maintenance every 4 months |
| Onasemnogene abeparvovec | IV infusion | Once |
| Risdiplam | Oral | Daily |
Statistics
The following are statistical data on the SMA treatments:
- Over 60% of patients receiving Nusinersen showed improvements in motor function.
- Long-term studies indicate that Zolgensma significantly enhances quality of life.
- Risdiplam has been reported to have a significant impact on survival rates in infants.
Mind Map of Treatment Options
Spinal Muscular Atrophy └── Treatments ├── Nusinersen │ ├── Mechanism: SMN2 modification │ ├── Administration: Intrathecal │ └── Patients: All ages ├── Onasemnogene abeparvovec │ ├── Mechanism: Gene therapy │ ├── Administration: IV │ └── Patients: < 2 years └── Risdiplam ├── Mechanism: SMN2 target ├── Administration: Oral └── Patients: All ages
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