Drug Pipeline

13 drugs associated with Congenital myotonic dystrophy type 1

DrugPhaseSponsor
Amlodipine

**Mechanism of action on blood pressure** Amlodipine is considered a peripheral arterial vasodilator that exerts its action directly on vascular smooth muscle to lead to a reduction in peripheral vascular resistance, causing a decrease in blood pressure. Amlodipine is a dihydropyridine calcium antagonist (calcium ion antagonist or slow-channel blocker) that inhibits the influx of calcium ions into both vascular smooth muscle and cardiac muscle. Experimental studies imply that amlodipine binds t

Phase 1University of Rochester
ERX-963
Phase 1Expansion Therapeutics, Inc.
PGN-EDODM1 for infusion
Phase 1PepGen Inc
Ranolazine

Myocardial ischemia exerts effects on adenosine triphosphate flux, leading to a decrease in the energy available for contraction and relaxation of the heart muscle. Electrolyte balance of sodium and potassium is necessary for maintaining normal cardiac contraction and relaxation. Disruption of adequate sodium and potassium electrolyte balance leads to excessively high concentrations of sodium and calcium, which likely interferes with oxygen supply to the heart muscle. This imbalance eventually l

Phase 1Ohio State University
ATX-01
Phase 2ARTHEx Biotech S.L.
rhIGF-I/rhIGFBP-3

Insulin-like growth factor I receptor agonist

Phase 2Insmed Incorporated
SAR446268
Phase 2Sanofi
SomatoKine/IPLEX
Phase 2University of Rochester
VX-670
Phase 2Vertex Pharmaceuticals Incorporated
AOC 1001 (del-desiran)
Phase 3Avidity Biosciences, Inc.
Del-desiran (AOC 1001)
Phase 3Avidity Biosciences, Inc.
DYNE-101
Phase 3Dyne Therapeutics
Methylphenidate

While its exact mechanism is unclear, methylphenidate (MPH) has been shown to act as a norepinephrine and dopamine reuptake inhibitor (NDRI), thereby increasing the presence of these neurotransmitters in the extraneuronal space and prolonging their action. There is a dose-related effect of psychostimulants on receptor stimulation, where higher doses are shown to increase norepinephrine (NE) and dopamine (DA) efflux throughout the brain which can result in impaired cognition and locomotor-activat

Phase 3Laval University