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Using Gene Editing to Correct DM

A potentially revolutionary technology may allow development of a drug for DM that can correct a patient’s DNA by selectively removing the expanded CTG and CCTG repeats in DM1 and DM2, respectively.

Adequate Rationale to Launch Clinical Trials

Clinical trials involve substantial investments by drug developers, but especially by DM patients and families. Clinical trials proposed for our community must have adequate scientific rationale behind them.

Changes in NINDS Postdoctoral Training Initiatives

MDF runs down some important changes in the purposes and rationale of NINDS funding mechanisms for postdoctoral fellows.

FDA: How to Talk with Us about INDs

FDA released an online course to accompany their draft guidance on how to best communicate with the Agency in preparing and filing an IND.

Gene Editing for DM

Gene editing is a potential avenue for therapy development in DM. With the safety, efficacy and delivery challenges, how do we get there?

Improving Clinical Trials in Myotonic Dystrophy: Thurman Wheeler, M.D.

When Dr. Thurman Wheeler was a resident in neurology, he remembers a senior physician telling him that myotonic dystrophy would probably be one of the most difficult diseases to treat because it involves so many body systems.

Treating Sleep Disorders in DM1

Sleep studies are indicated for DM1 patients with disrupted sleep or daytime sleepiness in order to direct selection of the optimal treatment regimen.

Is NINR a Source for DM Funding?

A new clinical trial planning grant initiative from the National Institute of Nursing Research (NINR) provides opportunities to develop an evidentiary basis for the consensus-driven clinical care recommendations currently under development at MDF for DM1.

AMPK/mTORC1 Signaling as a Therapeutic Target for DM1

Studies of AMPK/mTORC1 signaling in DM1 identify novel therapeutic targets for DM, and may offer an opportunity to repurpose approved drugs for both muscle and cognitive symptoms.

MDF Finished Year 10 with Flying Colors

2016 was a banner year for the Myotonic Dystrophy Foundation. We completed our 10th year driving Care and a Cure for people and families living with DM, wrapped up year 2 of a 3-year, $5M drug development acceleration effort and oversaw the largest expansion of our Care programming and resources ever. 

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