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.

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Here are some of the proposed locations for the study


United States, California
Stanford UniversityNot yet recruiting
Palo Alto, California, United States, 94304
Contact: Tia Nguyen    650-498-8771   
Principal Investigator: John Day, MD         
United States, Iowa
University of Iowa HospitalsNot yet recruiting
Iowa City, Iowa, United States, 52242
Contact: Diane Recker, BSN, RN    319-335-6073   
Principal Investigator: Katherine Mathews, MD         
United States, New York
University of Rochester Medical CenterNot yet recruiting
Rochester, New York, United States, 14642
Contact: James Hilbert    585-273-5590   
Contact: Jeanne Dekdebrun    585-276-4611   
Principal Investigator: Johanna Hamel, MD         
Sub-Investigator: Emma Ciafaloni, MD         
United States, Virginia
Children’s Specialty Group, PLLC, Division of Child & Adolescent Neurology, Children’s Hospital of the King’s DaughtersNot yet recruiting
Norfolk, Virginia, United States, 23510
Contact: Terrie Conklin, RN, CCRC    757-668-9356    terrie.conklin@CHKD.ORG   
Principal Investigator: Crystal Proud, MD         
Canada, Ontario
Children’s Hospital London Health Sciences Centre (LHSC)Not yet recruiting
London, Ontario, Canada, N6A4G5
Contact: Rhiannon Hicks    519-685-8441   
Principal Investigator: Craig Campbell, MD, MSc, FRCPC         

Sponsors and CollaboratorsAMO Pharma LimitedInvestigators

Study Director:Joseph P Horrigan, MDAMO Pharma
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