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%

Relaxation vs no treatment

Physical health: Reduction of bleedings, factor replacement, pain · adults and children with hemophilia

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 7 participants
Very low

Alglucosidase alfa vs placebo

Change from baseline in 6MWT (metres) · 90 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 90 participants
Moderate

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

Infusion reactions · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

Need for respiratory support · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

Use of a walking aid / wheelchair · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

QoL · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

Treatment‐related adverse events · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

Change from baseline in 6MWT (metres) · 13 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 5 participants
Very low

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

FVC (% predicted) Change from baseline · 13 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 5 participants
Very low

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

Infusion reactions · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

Need for respiratory support · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

Use of a walking aid / wheelchair · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa vs placebo

FVC (% predicted) Change from baseline · 90 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 90 participants
Moderate

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

QoL · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

Adverse events · 13 people with LOPD

It is very uncertain whether alglucosidase alfa plus albuterol changes adverse events compared with alglucosidase alfa plus placebo (very low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.67 [0.38, 1.18]
1 study · 13 participants
Very low

VAL‐1221 vs alglucosidase alfa

Change from baseline in 6MWT (metres) · adults with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 12 participants
Very low

VAL‐1221 vs alglucosidase alfa

FVC (% predicted) Change from baseline · adults with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

VAL‐1221 vs alglucosidase alfa

Infusion reactions · adults with LOPD

It is very uncertain whether VAL‐1221 changes infusion reactions compared with alglucosidase alfa (very low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
OR 2.8
1 study · 12 participants
Very low

VAL‐1221 vs alglucosidase alfa

Need for respiratory support · adults with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

VAL‐1221 vs alglucosidase alfa

Use of a walking aid / wheelchair · adults with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found