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RESEARCH PRODUCT
Myotonic dystrophy type 1 drug development: A pipeline toward the market
Marta Pascual-gilabertArturo Lopez-castelRuben Arterosubject
0301 basic medicineDrugmedia_common.quotation_subjectMyotonic dystrophyDiseaseBioinformaticsMarketing authorizationMyotonic dystrophy03 medical and health sciences0302 clinical medicineGene therapyDrug DevelopmentDrug DiscoveryMedicineAnimalsHumansAntisense oligonucleotideRepurposingmedia_commonPharmacologybusiness.industryRepurposing drugmedicine.diseaseClinical trialClinical trialDrug repositioning030104 developmental biologyDrug development030220 oncology & carcinogenesisbusinessPost-Screen (Grey)description
Highlights • Myotonic dystrophy, a neuromuscular disease, affects at least around half a million people worldwide. • Close to two dozen preclinical and clinical drug development programs active. • Drugs encompass new chemical entities, repurposing, oligonucleotide, and gene therapy. • Tideglusib, mexiletine, and metformin are close to reaching marketing authorization.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2021-07-01 | Drug Discovery Today |