Myotonic Dystrophy Researcher Receives Award

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    • The Barry M. Goldwater Scholarship and Excellence in Education Program was established by Congress in 1986 to honor Goldwater, who served 30 years in the U.S. Senate.

      The Barry M. Goldwater Scholarship and Excellence in Education Program was established by Congress in 1986 to honor Goldwater, who served 30 years in the U.S. Senate.

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  • Editor’s note: For more information, contact David Schug, National and International Scholarships Program director, 217-333-4710, email

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New Info about the AMO Myotonic Dystrophy Drug

Here is some information from the Muscular Dystrophy Group in the UK about the AMO drug that will be tested in the USA. I may be difficult to read if so click here for the link to the full report and look on page 9-10


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Cydan, Inc is using new business model to accelerate Myotonic Dystrophy drug

Developing a new drug to treat myotonic dystrophy is very expensive. It can take tens millions of dollars to make and test a new drug. Cydan a comapny has a new novel approach that spins off a new company and aquires capital n a novel way to help reduce the risk of this new drug development. For example Ionis Pharmaceuticals spents millions developing a drug that in fact they did not bring to market. A big loss this new framework might lead to more drug development for us! Below is a presentation on this technique


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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

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Another study advancing a potential treatment of myotonic Dystrophy

This is a fairly scientific study of using the CRISPER Technology to help find a treatment for myotonic dystrophy. DM1 is caused by expanded CTG repeats in the 30 UTR. It is conceivable that simply deleting the expanded CTG repeats may cure the disease. With the advancement of therapeutic genome-editing technologies, this is becoming more realistic.


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