Drisapersen
Drisapersen is an oligonucleotide pharmaceutical. It is currently being investigated in clinical studies.
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Clinical
Clinical Trials
10 clinical trials
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Indications Phases 4
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Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Muscular dystrophies | D009136 | HP_0003560 | G71.0 | 1 | 3 | 4 | — | 2 | 10 |
| Duchenne muscular dystrophy | D020388 | EFO_0000429 | — | — | 3 | 3 | — | 2 | 8 |
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Indications Without Phase
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Muscular diseases | D009135 | HP_0003198 | G72.9 | — | — | — | — | 1 | 1 |
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Drisapersen |
| INN | drisapersen |
| Description | Drisapersen (also known as Kyndrisa, PRO051 and GSK2402968) is an experimental drug that was under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. The drug is a 2'-O-methyl phosphorothioate oligonucleotide that alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA.
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| Classification | Oligonucleotide |
| Drug class | antisense oligonucleotides |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | 1251830-50-8 |
| RxCUI | — |
| ChEMBL ID | CHEMBL2108735 |
| ChEBI ID | — |
| PubChem CID | 121494119 |
| DrugBank | — |
| UNII ID | 73D586DAMF (ChemIDplus, GSRS) |
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Safety
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Adverse Events
0 adverse events reported
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