Findings

What do systematic reviews say?

2,261 questions · 2,280 findings · 114 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 856 · 37.5%GRADE not reported 58 · 2.5%Very low 550 · 24.1%Low 502 · 22%Moderate 271 · 11.9%High 43 · 1.9%

Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone

Resolution or reduction in severity of any other dental complication: reduction in severity of gingivitis (measured by BoP) · people with thalassaemia (any age)

1 finding from 1 review

Treatment of dental and orthodontic complications in thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 29 participants
Very low

Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone

Resolution or reduction in severity of any other dental complication: reduction in severity of periodontitis (measured by reduction in clinical attachment loss) · people with thalassaemia (any age)

The review found no eligible studies for this question.

1 finding from 1 review

Treatment of dental and orthodontic complications in thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone

Resolution or reduction in severity of any other dental complication: oral infections (measured by reduction in or resolution of lesions such as periapical cysts, abscesses, or space infections) · people with thalassaemia (any age)

The review found no eligible studies for this question.

1 finding from 1 review

Treatment of dental and orthodontic complications in thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone

Resolution or reduction in severity of any other dental complication: systemic infections (measured by number of episodes after treatment for dental complications under antibiotic prophylaxis) · people with thalassaemia (any age)

The review found no eligible studies for this question.

1 finding from 1 review

Treatment of dental and orthodontic complications in thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone

Any other adverse effects related to treatment · people with thalassaemia (any age)

The review found no eligible studies for this question.

1 finding from 1 review

Treatment of dental and orthodontic complications in thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

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