Alexander disease

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

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Show: 2 trials

Phase I

1 trial
  • Molefy Pharma S.L. Commercial
    IMP not specified
    2025-524480-21-00 1 country 2026 n=72
    Recruiting

Phase I (First in Human)

1 trial
  • Ionis Pharmaceuticals Inc. Commercial
    2'-O-(2-METHOXYETHYL)-D-RIBOSE ANTISENSE OLIGONUCLEOTIDE TARGETING GLIAL FIBRILLARY ACIDIC PROTEIN MESSENGER RIBONUCLEIC ACID
    2024-510603-11-00 2 countries 2021 n=15 Orphan
    Ended
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Molefy Pharma S.L. Commercial 1 Phase I 1
Ionis Pharmaceuticals Inc. Commercial 1 Phase I (First in Human) 0
New trials by year
2021
1
2026
1
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