Condition

Down syndrome

Clinical trials and treatment information for Down syndrome

1.6M

People Affected

Active Trials

400K

New Cases/Year

29K

Deaths/Year

Treatment Rankings

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

Thyroid Hormone Replacement (Levothyroxine) for Hypothyroidism

95% Effectiveness• 95% Confidence• 65% Safety• 100 trials• 10K participants

HIGH EvidenceExcellent ValueDose: 2-6 mcg/kg/day, titrated to TSH levels

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Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

2-4 weeks

Duration

lifetime

Response Rate

95%

Remission Rate

Number Needed to Treat (NNT)

Number Needed to Harm (NNH)

Common Side Effects:

Hyperactivity (from overdose): 5%

Insomnia (from overdose): 3%

Tremors (from overdose): 2%

Annual Cost of Care

Drug Cost

$100

Monitoring

$200

Side Effects

$50

Total Annual

$350

Cost-Effectiveness

EXCELLENT

QALYs Gained

1.5

Cost per Responder

$368.42

Treatment Outcomes

Efficacy Outcomes

Overall Effectiveness

+95%

Response Rate

+95%

Common Side Effects

Hyperactivity (from overdose)

+5%

Insomnia (from overdose)

+3%

Tremors (from overdose)

+2%

Clinical Trial Phases:

Phase 4

Surgical Repair of Congenital Heart Defects

90% Effectiveness• 95% Confidence• 60% Safety• 1 trials• 100K participants

HIGH EvidenceExcellent Value

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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 (post-recovery)

Duration

one-time surgery, followed by lifelong monitoring

Response Rate

90%

Remission Rate

85%

Common Side Effects:

Infection: 8%

Arrhythmias: 10%

Bleeding: 5%

Post-operative pain: 30%

Annual Cost of Care

Drug Cost

Monitoring

$1,000

Side Effects

$2,000

Total Annual

$3,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

Cost per Remission

$3,529.41

Cost per Responder

$3,333.33

Treatment Outcomes

Primary Outcomes

Systolic Pulmonary Artery Pressure60 mmHg

-50% (-30 mmHg)

Peripheral Oxygen Saturation (SpO2)85%

+14.1% (+12%)

Left Ventricular Ejection Fraction55%

+12.7% (+7%)

NT-proBNP Level500 pg/mL

-60% (-300 pg/mL)

Secondary Benefits

Weight-for-Age Z-score-1.5

+66.7% (+1.0)

Annual Episodes of Lower Respiratory Tract Infection4 episodes/year

-75% (-3 episodes/year)

Vineland Adaptive Behavior Scales (VABS) Daily Living Skills Score60/100

+13.3% (+8 points)

Common Side Effects

Infection

+8%

Arrhythmias

+10%

Bleeding

+5%

Clinical Trial Phases:

Phase 4

Early Intervention Programs (Comprehensive Developmental Therapies)

85% Effectiveness• 90% Confidence• 95% Safety• 500 trials• 50K participants

MODERATE EvidenceGood ValueDose: 5-15 hours/week of structured therapy/support

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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 (varying intensity)

Response Rate

80%

Remission Rate

Common Side Effects:

Fatigue: 5%

Temporary frustration: 3%

Annual Cost of Care

Drug Cost

Monitoring

$500

Side Effects

Total Annual

$500

Cost-Effectiveness

GOOD

QALYs Gained

Cost per Responder

$18,750

Treatment Outcomes

Efficacy Outcomes

Overall Effectiveness

+85%

Response Rate

+80%

Common Side Effects

Fatigue

+5%

Temporary frustration

+3%

Clinical Trial Phases:

Phase 4

Special Education Services

80% Effectiveness• 85% Confidence• 95% Safety• 300 trials• 100K participants

MODERATE EvidenceGood ValueDose: 20-30 hours/week of specialized instruction/support

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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

Duration

childhood through young adulthood

Response Rate

75%

Remission Rate

Common Side Effects:

Frustration with learning: 5%

Social stigma: 2%

Annual Cost of Care

Drug Cost

Monitoring

$500

Side Effects

Total Annual

$500

Cost-Effectiveness

GOOD

QALYs Gained

Cost per Responder

$24,000

Treatment Outcomes

Efficacy Outcomes

Overall Effectiveness

+80%

Response Rate

+75%

Common Side Effects

Frustration with learning

+5%

Social stigma

+2%

Clinical Trial Phases:

Phase 4

Speech-Language Therapy

75% Effectiveness• 85% Confidence• 95% Safety• 5 trials• 20K participants

MODERATE EvidenceGood ValueDose: 1-3 sessions/week (30-60 min/session)

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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

long-term (varying intensity)

Response Rate

70%

Remission Rate

Common Side Effects:

Fatigue: 3%

Frustration: 2%

Annual Cost of Care

Drug Cost

Monitoring

$500

Side Effects

Total Annual

$500

Cost-Effectiveness

GOOD

QALYs Gained

0.5

Cost per Responder

$7,142.86

Treatment Outcomes

Primary Outcomes

Speech Intelligibility30% intelligible (to unfamiliar listeners)

+50% (+15% (absolute increase))

Expressive Vocabulary Standard Score60 points (on a standardized test, mean=100, SD=15)

+16.67% (+10 points)

Mean Length of Utterance (MLU) in morphemes1.5 morphemes

+40% (+0.6 morphemes)

Receptive Language Standard Score65 points (on a standardized test, mean=100, SD=15)

+12.31% (+8 points)

Secondary Benefits

Social Communication Skills Standard Score70 points (on a parent-report scale, mean=100, SD=15)

+10% (+7 points)

Functional Communication Independent Acts5 independent communicative acts per hour

+60% (+3 independent communicative acts per hour)

Parental Stress (Communication-Related) Scale Score30 points (on a 0-60 scale, higher is more stress)

-23.33% (-7 points)

Common Side Effects

Fatigue

+3%

Frustration

+2%

Clinical Trial Phases:

Phase 4

Physical Therapy

70% Effectiveness• 80% Confidence• 95% Safety• 55 trials• 15K participants

MODERATE EvidenceGood ValueDose: 1-2 sessions/week (30-60 min/session)

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

3-6 months

Duration

long-term (varying intensity)

Response Rate

65%

Remission Rate

Common Side Effects:

Muscle soreness: 5%

Fatigue: 4%

Annual Cost of Care

Drug Cost

Monitoring

$500

Side Effects

Total Annual

$500

Cost-Effectiveness

GOOD

QALYs Gained

0.4

Cost per Responder

$6,153.85

Treatment Outcomes

Primary Outcomes

Gross Motor Function Measure (GMFM-88) Total Score65% of max score (for toddlers/preschoolers with Down syndrome)

+12% (+7.8 percentage points)

Peabody Developmental Motor Scales-2 (PDMS-2) Gross Motor Quotient (GMQ)60 points (age-equivalent for young children with Down syndrome)

+10% (+6 points)

Pediatric Balance Scale (PBS) Score45/56 points (for school-aged children with Down syndrome)

+8% (+3.6 points)

Secondary Benefits

Pediatric Quality of Life Inventory (PedsQL) - Physical Functioning Scale Score (Parent Proxy)60/100 points (for children with Down syndrome)

+10% (+6 points)

6-Minute Walk Test (6MWT) Distance250 meters (for adolescents/adults with Down syndrome)

+15% (+37.5 meters)

Adaptive Behavior Assessment System-3 (ABAS-3) - Motor Domain Standard Score65 points (low average for functional motor skills in individuals with Down syndrome)

+8% (+5.2 points)

Common Side Effects

Muscle soreness

+5%

Fatigue

+4%

Clinical Trial Phases:

Phase 4

Occupational Therapy

70% Effectiveness• 80% Confidence• 95% Safety• 1 trials• 15K participants

MODERATE EvidenceGood ValueDose: 1-2 sessions/week (30-60 min/session)

Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️

Meth💀

Cigarettes🚬

Chemo☢️

Alcohol🍺

Morphine💊

Antibiotics💉

Tylenol💊

Exercise🏃

Water💧

DangerousModerateSafe

Time to Effect

3-6 months

Duration

long-term (varying intensity)

Response Rate

65%

Remission Rate

Common Side Effects:

Frustration: 3%

Fatigue: 2%

Annual Cost of Care

Drug Cost

Monitoring

$500

Side Effects

Total Annual

$500

Cost-Effectiveness

GOOD

QALYs Gained

0.4

Cost per Responder

$6,153.85

Treatment Outcomes

Primary Outcomes

Adaptive Behavior (Daily Living Skills)Vineland Adaptive Behavior Scales (VABS-3), Daily Living Skills domain: 65 (standard score)

+7.7% (+5 standard score points)

Fine Motor SkillsBruininks-Oseretsky Test of Motor Proficiency (BOT-2), Fine Motor Control composite: 35 (standard score)

+14.3% (+5 standard score points)

Gross Motor SkillsBruininks-Oseretsky Test of Motor Proficiency (BOT-2), Gross Motor Composite: 38 (standard score)

+13.2% (+5 standard score points)

Participation in Play/School ActivitiesPediatric Evaluation of Disability Inventory - Computer Adaptive Test (PEDI-CAT), Participation domain: 20 (scaled score)

+25% (+5 scaled score points)

Secondary Benefits

Caregiver Assistance Required for Daily TasksPediatric Evaluation of Disability Inventory (PEDI), Self-Care Functional Skills score: 25/100 (lower score indicates more assistance needed)

+40% (+10 points on PEDI Functional Skills score)

Social ParticipationPediatric Evaluation of Disability Inventory - Computer Adaptive Test (PEDI-CAT), Social/Cognitive domain: 22 (scaled score)

+22.7% (+5 scaled score points)

Sensory Processing DifficultiesSensory Profile 2, Overall Sensory Processing Score: 70 (higher score indicates more difficulties)

-7.1% (-5 standard score points)

Common Side Effects

Frustration

+3%

Fatigue

+2%

Clinical Trial Phases:

Phase 4