Angelman's syndrome

4 trials 3 recruiting MedDRA PT
4 Registered trials
3 Recruiting
3 Unique sponsors
6 Unique IMPs
4 Commercial trials
Drug classes (ATC)
— ATC code not specified 4
Mechanism of action (INN-stem analysis)
Unknown / small molecule 4 substances
Antisense oligonucleotide 2 substances
Phase III: 2 Phase I (Other): 1 Phase I (First in Human): 1

Filter these 4 trials by country, sponsor, date range and more.

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Show: 4 trials

Phase III

2 trials
  • Ultragenyx Pharmaceutical Inc. Commercial
    APAZUNERSEN; CALCIUM CHLORIDE, GLUCOSE, ANHYDROUS, SODIUM HYDROGEN CARBONATE, POTASSIUM CHLORIDE, SODIUM CHLORIDE, SODIUM PHOSPHATE, MAGNESIUM SULFATE; SODIUM DIHYDROGEN PHOSPHATE DIHYDRATE, DISODIUM PHOSPHATE, POTASSIUM CHLORIDE, SODIUM CHLORIDE, CALCIUM CHLORIDE DIHYDRATE, MAGNESIUM SULFATE HEPTAHYDRATE
    2024-510917-14-00 3 countries 2024 n=38 Orphan
    Recruiting
  • Ultragenyx Pharmaceutical Inc. Commercial
    2'-O, 4'-C-METHYLENE-P-THIO-ADENYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-P-THIOGUANYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-P-THIO-ADENYLYL-(3'->5')-2'-DEOXY-P-THIOADENYLYL-(3'->5')-2'-DEOXY-P-THIO-THYMIDYLYL-(3'->5')-2'-DEOXY-P-THIO-GUANYLYL-(3'->5')-2'-DEOXY-P-THIO-GUANYLYL-(3'->5')-2'-DEOXY-P-THIO-CYTIDYLYL-(3'->5')-2'-DEOXY-P-THIO-ADENYLYL-(3'->5')-2'-DEOXY-P-THIO-CYTIDYLYL-(3'->5')-2'-DEOXY-P-THIO-ADENYLYL-(3'->5')-2'-DEOXY-P-THIO-THYMIDYLYL-(3'->5')-2'-DEOXY-P-THIO-CYTIDYLYL-(3'->5')-2'-DEOXY-P-THIO-THYMIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-5-METHYL-P-THIO-CYTIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-5-METHYL-P-THIO-URIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENE-5-METHYL-P-THIO-URIDYLYL-(3'->5')-2'-O, 4'-C-METHYLENEGUANOSINE; SODIUM DIHYDROGEN PHOSPHATE DIHYDRATE, DISODIUM PHOSPHATE, POTASSIUM CHLORIDE, SODIUM CHLORIDE, CALCIUM CHLORIDE DIHYDRATE, MAGNESIUM SULFATE HEPTAHYDRATE
    2024-512600-19-00 4 countries 2025 n=40 Orphan
    Recruiting

Phase I (Other)

1 trial
  • F. Hoffmann-La Roche AG Commercial
    RUGONERSEN
    2024-514797-45-00 3 countries 2020 n=28 Orphan
    Ended

Phase I (First in Human)

1 trial
  • Ionis Pharmaceuticals Inc. Commercial
    2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING UBE3A ANTISENSE TRANSCRIPT RNA
    2024-512589-32-00 2 countries 2024 n=8 Orphan
    Recruiting
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Ultragenyx Pharmaceutical Inc. Commercial 2 Phase III 2
F. Hoffmann-La Roche AG Commercial 1 Phase I (Other) 0
Ionis Pharmaceuticals Inc. Commercial 1 Phase I (First in Human) 1
New trials by year
2020
1
2024
2
2025
1
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