Findings

What do systematic reviews say?

632 questions · 636 findings · 33 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 197 · 31%GRADE not reported 13 · 2%Very low 133 · 20.9%Low 159 · 25%Moderate 121 · 19%High 13 · 2%

Intervention not reported

Mean percentage change from baseline in FVC (% predicted) · 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

Mean change from baseline in 6MWT (metres) · 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

Infusion‐related reactions (number of participants with one or more reaction) · adults and children with mucopolysaccharidosis I

The intervention showed no clear difference in infusion‐related reactions (number of participants with one or more… (low certainty).

1 finding from 1 review

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I 2019 · Cochrane Headline outcomes only
OR 0.51 [0.15, 1.71]
1 study · 45 participants
Low

Intervention not reported

Antibody production: number of participants developing IgG antibodies · adults and children with mucopolysaccharidosis I

It is very uncertain whether the intervention changes antibody production: number of participants developing IgG antibodies (very low certainty).

1 finding from 1 review

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I 2019 · Cochrane Headline outcomes only
OR 385.4 [17.47, 8500.51]
1 study · 45 participants
Very low

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

Antifibrinolytic agents (tranexamic acid (TXA) or epsilon aminocaproic acid (EACA)) vs Placebo, no intervention, or usual care

Outcome not reported · People on continuous direct oral anticoagulant (DOAC) treatment undergoing oral or dental procedures

The review found no eligible studies for this question.

1 finding from 1 review

Antifibrinolytic therapy for preventing oral bleeding in people on anticoagulants undergoing minor oral surgery or… 2018 · Cochrane All outcomes
no estimate extracted
0 studies
No studies found

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