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Miglustat

Pompe Disease

Amicus TherapeuticsSmall moleculePhase 1Active
CC
43/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
28/ 100
Competitive Position
89/ 100

Competitive context

8 Active assets in the Pompe Disease cohort.

Asset / SponsorPhaseRating
Avalglucosidase Alfa
Genzyme, a Sanofi Company
Phase 4A
Alglucosidase Alfa
Erasmus Medical Center
Phase 4A
Avalglucosidase Alfa
Genzyme, a Sanofi Company
Phase 4A
Albuterol
National Taiwan University Hospital
Phase 4BBB
IMMUNOGLOBULIN G 1 (HUMAN-MOUSE MONOCLONAL IDEC-C2B8 .GAMMA.1-CHAIN ANTI-HUMAN ANTIGEN CD 20), DISULFIDE WITH HUMAN-MOUSE MONOCLONAL IDEC-C2B8 .KAPPA.-CHAIN, DIMER
University of Florida
Phase 4BB

View all 28 →

Same molecule, other indications

Miglustat is also rated in:

Niemann-Pick disease type CDormant
BBB
Indicative ·
76 / 100
View asset →
Gaucher disease type IDormant
BB
Indicative ·
64 / 100
View asset →
cystic fibrosisDormant
B
Indicative ·
63 / 100
View asset →

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