Condition

Motor neuron disease

Clinical trials and treatment information for Motor neuron disease

273K

People Affected

Active Trials

22K

New Cases/Year

39K

Deaths/Year

Treatment Rankings

Ranked by evidence volume first. Medicine has spent centuries ranking things by vibes. This is an improvement.

Non-Invasive Ventilation (NIV)

90% Effectiveness• 95% Confidence• 85% Safety• 39 trials• 3K participants

HIGH EvidenceExcellent ValueDose: Titrated positive airway pressure based on patient need

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Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

Immediate for symptom relief, months for survival benefit

Duration

Lifetime

Response Rate

70%

Remission Rate

Number Needed to Treat (NNT)

Number Needed to Harm (NNH)

Common Side Effects:

Facial skin irritation: 30%

Dry mouth: 20%

Bloating/gastric distension: 15%

Mask discomfort: 10%

Annual Cost of Care

Drug Cost

Monitoring

$2,800

Side Effects

$200

Total Annual

$3,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.5

ICER

$20,000/QALY

Cost per Responder

$4,286

Treatment Outcomes

Primary Outcomes

Time to Tracheostomy or Death12 months (without NIV)

+50% (+6 months)

Average Nocturnal Oxygen Saturation (SpO2)88% saturation

+4.5% (+4% saturation)

Dyspnea Severity (mMRC Scale)mMRC score: 2/4

-25% (-0.5 points)

Morning Arterial PCO250 mmHg

-10% (-5 mmHg)

Sleep Quality (PSQI Score)PSQI Score: 12/21

-33.3% (-4 points)

Secondary Benefits

Quality of Life (ALSAQ-40 Score)ALSAQ-40 score: 50/100

-10% (-5 points)

Respiratory Hospitalizations per Year1.5 hospitalizations/year

-66.7% (-1.0 hospitalization/year)

ALSFRS-R Score Decline Rate1.0 point/month decline

-50% (-0.5 points/month)

Common Side Effects

Facial skin irritation

+30%

Dry mouth

+20%

Bloating/gastric distension

+15%

Clinical Trial Phases:

Phase 4

Gastrostomy (PEG Tube)

85% Effectiveness• 90% Confidence• 70% Safety• 5 trials• 2.5K participants

HIGH EvidenceExcellent ValueDose: N/A (procedural)

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Comparative Safety Scale(Higher is safer)

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Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

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DangerousModerateSafe

Time to Effect

Immediate for nutritional support, long-term for aspiration prevention and quality of life

Duration

Lifetime

Response Rate

90%

Remission Rate

Number Needed to Treat (NNT)

Number Needed to Harm (NNH)

Common Side Effects:

Local infection: 7%

Leakage at insertion site: 7%

Pain: 5%

Tube displacement: 3%

Annual Cost of Care

Drug Cost

Monitoring

$2,000

Side Effects

$500

Total Annual

$2,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.3

ICER

$35,000/QALY

Cost per Responder

$2,778

Treatment Outcomes

Primary Outcomes

Body Weight60 kg

+2.5% (+1.5 kg)

Daily Caloric Intake1500 kcal/day

+50% (+750 kcal/day)

Serum Albumin3.2 g/dL

+15.625% (+0.5 g/dL)

Secondary Benefits

Caregiver Burden (Zarit Burden Interview score)45 points (on a 0-88 scale)

-22.2% (-10 points)

Fatigue Severity (Fatigue Severity Scale score)6.0 points (on a 1-7 scale, higher is worse)

-25% (-1.5 points)

Quality of Life (Nutrition domain score)40 points (on a 0-100 scale, higher is better)

+25% (+10 points)

Common Side Effects

Local infection

+7%

Leakage at insertion site

+7%

Pain

+5%

Clinical Trial Phases:

Phase 4

Tofersen (for SOD1-ALS)

60% Effectiveness• 80% Confidence• 55% Safety• 1 trials• 200 participants

MODERATE EvidencePoor ValueDose: 100 mg intrathecal injection every 4 weeks after loading doses

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Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

Weeks to months for biomarker changes, longer for clinical effect

Duration

Lifetime

Response Rate

Remission Rate

Common Side Effects:

CSF pleocytosis: 36%

Myelitis: 2%

Radiculitis: 2%

Papilledema: 1%

Annual Cost of Care

Drug Cost

$160,000

Monitoring

$10,000

Side Effects

$2,000

Total Annual

$172,000

Cost-Effectiveness

POOR

QALYs Gained

0.08

ICER

$1,000,000/QALY

Treatment Outcomes

Primary Outcomes

ALS Functional Rating Scale-Revised (ALSFRS-R)ALSFRS-R score: 38/48 points

-15.26% (-5.8 points over 28 weeks (slowing of decline))

Plasma Neurofilament Light Chain (NfL)110 pg/mL (plasma)

-45% (-49.5 pg/mL)

Slow Vital Capacity (SVC)80% of predicted

-6% (-4.8 percentage points of predicted over 28 weeks (slowing of decline))

Secondary Benefits

CSF SOD1 Protein Concentration70 ng/mL (CSF)

-35% (-24.5 ng/mL)

Muscle Strength (HHD total score)65 points (sum of 16 muscles, HHD total score)

-3.08% (-2 points over 28 weeks (slowing of decline))

Common Side Effects

CSF pleocytosis

+36%

Myelitis

+2%

Radiculitis

+2%

Clinical Trial Phases:

Phase 3Phase 4

Edaravone

55% Effectiveness• 85% Confidence• 65% Safety• 11 trials• 600 participants

HIGH EvidencePoor ValueDose: 60 mg IV infusion daily for 14 days, followed by 14-day break; subsequent cycles 10 of 14 days, 14-day break (Oral suspension also available)

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Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

3-6 months

Duration

Lifetime (cycles)

Response Rate

Remission Rate

Number Needed to Treat (NNT)

Number Needed to Harm (NNH)

Common Side Effects:

Contusion: 9.6%

Gait disturbance: 8.4%

Headache: 7.2%

Infusion site reaction: 7%

Annual Cost of Care

Drug Cost

$165,000

Monitoring

$7,500

Side Effects

$500

Total Annual

$173,000

Cost-Effectiveness

POOR

QALYs Gained

0.07

ICER

$500,000/QALY

Treatment Outcomes

Primary Outcomes

ALS Functional Rating Scale-Revised (ALSFRS-R) score change38/48 points

-12.6% (-4.8 points over 24 weeks)

Rate of ALSFRS-R decline1.2 points/month

-33% (-0.4 points/month)

Forced Vital Capacity (FVC) decline85% predicted

-2% (-1.7% predicted over 24 weeks)

Secondary Benefits

Urinary 8-hydroxy-2'-deoxyguanosine (8-OHdG) levels15 ng/mg creatinine

-20% (-3 ng/mg creatinine)

Quadriceps Muscle Strength (Dynamometry)50 lbs

-4% (-2 lbs over 24 weeks)

Common Side Effects

Contusion

+9.6%

Gait disturbance

+8.4%

Headache

+7.2%

Clinical Trial Phases:

Phase 3Phase 4

Riluzole

45% Effectiveness• 90% Confidence• 65% Safety• 43 trials• 2K participants

HIGH EvidenceGood ValueDose: 50 mg twice daily

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Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

6-12 months for survival benefit

Duration

Lifetime

Response Rate

Remission Rate

Number Needed to Treat (NNT)

Number Needed to Harm (NNH)

Common Side Effects:

Nausea: 20%

Asthenia: 14%

Liver enzyme elevation (>3x ULN): 15%

Dizziness: 11%

Annual Cost of Care

Drug Cost

$2,000

Monitoring

$200

Side Effects

$100

Total Annual

$2,300

Cost-Effectiveness

GOOD

QALYs Gained

0.1

ICER

$80,000/QALY

Treatment Outcomes

Primary Outcomes

Median Survival Time14 months (untreated median)

+14.3% (+2 months)

Rate of ALSFRS-R Score Decline1.1 points/month (untreated)

-15% (-0.16 points/month)

Tracheostomy-free Survival20 months (untreated median)

+15% (+3 months)

Secondary Benefits

Rate of Forced Vital Capacity (FVC) Decline1.8% of predicted per month (untreated)

-8% (-0.14% of predicted per month)

Plasma Neurofilament Light Chain (NfL)50 pg/mL (elevated in ALS)

-15% (-7.5 pg/mL)

Common Side Effects

Nausea

+20%

Asthenia

+14%

Liver enzyme elevation (>3x ULN)

+15%

Clinical Trial Phases:

Phase 3Phase 4