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-12, NP031112) 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
Here are some of the proposed locations for the study
Locations
United States, California | |
Stanford University | Not yet recruiting |
Palo Alto, California, United States, 94304 | |
Contact: Tia Nguyen 650-498-8771 tinguyen@stanford.edu | |
Principal Investigator: John Day, MD | |
United States, Iowa | |
University of Iowa Hospitals | Not yet recruiting |
Iowa City, Iowa, United States, 52242 | |
Contact: Diane Recker, BSN, RN 319-335-6073 diane-recker@uiowa.edu | |
Principal Investigator: Katherine Mathews, MD | |
United States, New York | |
University of Rochester Medical Center | Not yet recruiting |
Rochester, New York, United States, 14642 | |
Contact: James Hilbert 585-273-5590 james_hilbert@URMC.Rochester.edu | |
Contact: Jeanne Dekdebrun 585-276-4611 jeanne_Dekdebrun@URMC.Rochester.edu | |
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 Daughters | Not 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 rhiannon.hicks@lhsc.on.ca | |
Principal Investigator: Craig Campbell, MD, MSc, FRCPC |
Sponsors and CollaboratorsAMO Pharma LimitedInvestigators
Study Director: | Joseph P Horrigan, MD | AMO Pharma |