New drug, Tideglusib, approach moving to clinical trial shortly

There is a new compound that is being tested to see if it can help with congenital myotonic dystrophy. It is being tested for a number of applications including tooth repair and Alzheimer’s  and just might help with the congenital form of myotonic dsytrophy. this is a molecule being developed by AMO pharma.

Tideglusib (NP-12NP031112) is a potent, selective and irreversible[1] small molecule non-ATP-competitive glycogen synthase kinase 3 (GSK-3) inhibitor.

Potential applications[edit]

Tideglusib is under investigation for multiple applications:

  • Alzheimer’s disease and progressive supranuclear palsy. As of 2017 it was undergoing Phase IIa[2] and IIb clinical trials.[3][4][5][6] The first trial to be published (in English) was Phase IIand demonstrated that tideglusib was well tolerated, except for some moderate, asymptomatic, fully reversible increases in liver enzymes.[4]
  • Tooth repair mechanisms that promotes dentine reinforcement of a sponge structure until the sponge biodegrades, leaving a solid dentine structure. In 2016, the results of animal studies were reported in which 0.14 mm holes in mouse teeth were permanently filled.[7]
  • Tideglusib is being studied in Phase II clinical trials as a treatment for congenital/juvenile-onset myotonic muscular dystrophy type I.[8]

There is a clinical study that will be starting shortly and you might be able to participate when this trial opens. Click here for more information.

https://clinicaltrials.gov/ct2/show/NCT03692312?cond=Myotonic+Dystrophy%2C+Congenital&rank=1

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Continue reading

Full Phase I Drug Trial Recruiting for Myotonic Dystrophy (DM1)

This week ISIS pharmaceuticals announced the commencement of recruiting for Phase I of the Myotonic Dystrophy Drug Trial. This is tremendous news for the myotonic dystrophy community. The promise of scientific research to define the DNA site, identify the cause of the disease,  then begin to develop treatments that would reverse the problems in the cell are culminating  to the promise of hope for all that suffer from this horrible affliction.

If you want to participate in this study you must act quickly and contact the centers that are conducting the research. Find the closest location and contact the person via email or phone. You must meet all (meaning all) the qualifications for the trial. The full information is on CLINICALTRIALS

Continue reading