Drug Pipeline
2 drugs associated with Congenital myotonic dystrophy type 1 (Steinert disease, congenital-onset DM1)
| Drug | Phase | Sponsor | |
|---|---|---|---|
IONIS-DMPKRx Amiloride-sensitive sodium channel, ENaC mRNA antisense inhibitor | Phase 2 | Ionis Pharmaceuticals, Inc. | ↗ |
Tideglusib GSK-3 is a proline/serine protein kinase that is ubiquitously expressed and involved in many cellular signaling pathways. From all its diverse functions, it plays a key role in Alzheimer's disease. This role is related to its link with β-amyloid and tau pathology. It has been suggested that aberrant Wnt or insulin signaling results in increased GSK-3 function. This kinase acts on γ-secretase producing the hyperphosphorylation of tau, the formation of neurofibrillary tangles and senile plaques. T | Phase 3 | AMO Pharma Limited | ↗ |