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 is under investigation for multiple applications:
- Alzheimer’s disease and progressive supranuclear palsy. As of 2017 it was undergoing Phase IIa and IIb clinical trials. 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.
- 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.
- Tideglusib is being studied in Phase II clinical trials as a treatment for congenital/juvenile-onset myotonic muscular dystrophy type I.
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 University||Not yet recruiting|
|Palo Alto, California, United States, 94304|
|Contact: Tia Nguyen 650-498-8771 firstname.lastname@example.org|
|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 email@example.com|
|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|
|Children’s Hospital London Health Sciences Centre (LHSC)||Not yet recruiting|
|London, Ontario, Canada, N6A4G5|
|Contact: Rhiannon Hicks 519-685-8441 firstname.lastname@example.org|
|Principal Investigator: Craig Campbell, MD, MSc, FRCPC|
Sponsors and CollaboratorsAMO Pharma LimitedInvestigators
|Study Director:||Joseph P Horrigan, MD||AMO Pharma|