Date: 2014-06-09
Type of information: Initiation of the trial
phase: 1
Announcement: initiation of the study
Company: Isis Pharmaceuticals (USA)
Product: ISIS-DMPK Rx
Action
mechanism: ISIS-DMPKRx is an antisense drug that has been designed to correct the underlying genetic defect that causes Myotonic Dystrophy Type 1. The disease is caused by a genetic defect in the dystrophia myotonica-protein kinase, or DMPK, gene in which a sequence of three nucleotides, CTG, repeats extensively. This DNA expansion produces an abnormally large toxic RNA that accumulates in cells, including muscle cells, and prevents production of proteins essential for normal cellular function. In addition to disabling muscle spasms and progressive muscle wasting and weakness, DM1 also affects many other organs within the body.
Disease: myotonic dystrophy type 1
Therapeutic area: Rare diseases
Country:
Trial details:
Latest
news: * On June 9, 2014, Isis Pharmaceuticals announced that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement.
