Findings

What do systematic reviews say?

668 questions · 685 findings · 68 reviews · Headline outcomes only

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No studies found 181 · 26.4%GRADE not reported 16 · 2.3%Very low 151 · 22%Low 211 · 30.8%Moderate 98 · 14.3%High 28 · 4.1%

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

VAL‐1221 vs alglucosidase alfa

QoL · 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

Treatment‐related adverse events · 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