Tofersen (for SOD1-ALS)
Treatment for Motor Neuron Disease
Typical Dosage: 100 mg intrathecal injection every 4 weeks after loading doses
Effectiveness
60%
Safety Score
55%
Clinical Trials
1
Participants
200
Comparative Safety Scale(Higher is safer)
Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
55
DangerousModerateSafe
Treatment Details
Dosage Range
100 mg intrathecal injection every 4 weeks after loading doses
Time to Effect
Weeks to months for biomarker changes, longer for clinical effect
Treatment Duration
Lifetime
Evidence Quality
MODERATEConfidence Score
80%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$160,000
Monitoring:$10,000
Side Effect Mgmt:$2,000
Total Annual:$172,000
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POORICER
$1,000,000/QALY
QALYs Gained
0.08
Comparison vs Best Supportive Care
Cost Difference
+$172,000/year
More expensive
QALY Difference
+0.08 QALYs
Better outcomes
Dominance
No dominance
Tofersen (for SOD1-ALS) Outcomes
for Motor Neuron Disease
Efficacy Outcomes
Overall Effectiveness
+60%
Common Side Effects
CSF pleocytosis
+36%
Myelitis
+2%
Radiculitis
+2%
Papilledema
+1%
Sources:
Active Clinical Trials
2 active trials recruiting for Tofersen (for SOD1-ALS) in Motor Neuron Disease
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)
NCT07259980NOT YET RECRUITING
69 participants
OBSERVATIONAL
Started: Jan 19, 2026
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
NCT07223723NOT YET RECRUITINGPHASE4
12 participants
INTERVENTIONAL
Started: Dec 2, 2025