Mucopolysaccharidosis III

3 trials 2 recruiting MedDRA PT
3 Registered trials
2 Recruiting
2 Unique sponsors
2 Unique IMPs
3 Commercial trials
Drug classes (ATC)
— ATC code not specified 3
Mechanism of action (INN-stem analysis)
Unknown / small molecule 2 substances
Phase III: 1 Phase I: 1 Phase I (First in Human): 1

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Show: 3 trials

Phase III

1 trial
  • Ultragenyx Pharmaceutical Inc. Commercial
    REBISUFLIGENE ETISPARVOVEC
    2023-510392-66-00 1 country 2020 n=24 Orphan
    Recruiting

Phase I

1 trial
  • Jcr Pharmaceuticals Co. Ltd. Commercial
    N-SULFOGLUCOSAMINE SULFOHYDROLASE FUSED TO A HUMANISED MONOCLONAL ANTIBODY TARGETING HUMAN TRANSFERRIN RECEPTOR
    2024-517045-14-00 1 country 2023 n=15 Orphan
    Ended

Phase I (First in Human)

1 trial
  • Ultragenyx Pharmaceutical Inc. Commercial
    REBISUFLIGENE ETISPARVOVEC
    2023-510032-37-00 1 country 2017 n=21 Orphan
    Recruiting
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Ultragenyx Pharmaceutical Inc. Commercial 2 Phase III, Phase I (First in Human) 2
Jcr Pharmaceuticals Co. Ltd. Commercial 1 Phase I 0
New trials by year
2020
1
2023
1
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