What do systematic reviews say?

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

Cipaglucosidase plus miglustat showed no clear difference in treatment‐related AEs: total treatment‐emergent adverse events… compared with alglucosidase alfa plus placebo (low certainty).

• Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
  RR 0.83 [0.49, 1.4]
  1 study · 123 participants
  Low

Avalglucosidase alfa vs alglucosidase alfa

Avalglucosidase alfa showed no clear difference in treatment‐related AEs: total treatment‐emergent adverse events… compared with alglucosidase alfa (low certainty).

• Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
  RR 0.92 [0.61, 1.4]
  1 study · 100 participants
  Low

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  2 studies · 1108 participants
  High

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 204 participants
  Low

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 61 participants
  Low

Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  4 studies · 634 participants
  Moderate

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  3 studies · 603 participants
  Moderate

Summary of findings ‐ monotherapy: lumacaftor vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  2 studies · 115 participants
  Very low

Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 55 participants
  Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 76 participants
  Low

Summary of findings ‐ ivacaftor vs placebo

• Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
  no estimate extracted
  3 studies · 232 participants
  Low

Summary of findings ‐ ivacaftor vs placebo

• Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 69 participants
  Low

Summary of findings ‐ ivacaftor vs placebo

• Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 140 participants
  Low

Summary of findings ‐ monotherapy: cavosonstat vs placebo

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 26 participants
  Very low

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor showed no clear difference in adverse events: number of participants with treatment‐emergent… compared with control (ivacaftor alone or tezacaftor plus ivacaftor) (moderate certainty).

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  OR 1.04 [0.53, 2.04]
  1 study · 258 participants
  Moderate

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

• Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
  no estimate extracted
  8 studies · 1057 participants
  Moderate