Findings

What do systematic reviews say?

167 questions · 167 findings · 8 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 80 · 47.9%GRADE not reported 1 · 0.6%Very low 45 · 26.9%Low 34 · 20.4%Moderate 7 · 4.2%High 0 · 0%

Intervention not reported

Quality of life · adults and children with mucopolysaccharidosis I

1 finding from 1 review

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 45 participants
Low

Intervention not reported

Mortality: number of deaths · adults and children with mucopolysaccharidosis I

1 finding from 1 review

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 45 participants
Low

Intervention not reported

Cardiac function · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Time to ventilation · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Survival · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
61
1 study · 18 participants
Low

Intervention not reported

Motor development · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Quality of life · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
GRADE not reported

Intervention not reported

Infusion‐related events · infantile‐onset Pompe disease

The intervention showed no clear difference in infusion‐related events (low certainty).

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
RR 0.83 [0.4, 1.76]
1 study · 18 participants
Low

Intervention not reported

Other adverse events · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Changes in globotriaosylceramide (Gb3) concentration in plasma · Anderson‐Fabry disease

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 39 participants
Very low

Intervention not reported

Renal function events (ITT population) · Anderson‐Fabry disease

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 82 participants
Very low

Intervention not reported

Symptoms and complications of disease: cardiac events · Anderson‐Fabry disease

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 82 participants
Very low

Intervention not reported

Symptoms and complications of disease: cerebrovascular events (ITT population) · Anderson‐Fabry disease

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 82 participants
Very low

Intervention not reported

Changes in globotriaosylceramide (Gb3) concentration in plasma and tissue · Anderson‐Fabry disease

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

Death · Anderson‐Fabry disease

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 36 participants
Very low

Intervention not reported

Pain Acroparaesthesia and Fabry crises · Anderson‐Fabry disease

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

Renal function · Anderson‐Fabry disease

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

Symptoms and complications of disease: cardiac events · Anderson‐Fabry disease

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 29 participants
Very low

Intervention not reported

Symptoms and complications of disease: cerebrovascular events · Anderson‐Fabry disease

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

Death · Anderson‐Fabry disease

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for Anderson‐Fabry disease 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found