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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.

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.

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. 

Wyck Foundation and MDF Grant Awards

In partnership with MDF, the Wyck Foundation awarded a number of new DM research grants.

Research Fellow Profile: Lukasz Sznajder

Lukasz Sznajder, Ph.D., is developing a mouse model for type 2 myotonic dystrophy, a crucial step that is expected to advance understanding of and therapy for this disease.

New Grants Awarded

MDF and the Wyck Foundation have awarded new grants to develop phenotypic mouse models of DM as well as new DM1 and DM2 cell lines. Read more about these new partnerships.

MDF Featured in The San Francisco Business Times

We are the focus of the latest “BioFlash" podcast by the San Francisco Business Times! Check out the interviews with our incredible champions, Erica and Jeremy Kelly (Chairman of the MDF Board), and our CEO, Molly White. We are thrilled by this opportunity to raise awareness of DM in the biotech community. Click here to read the article and listen to the podcast.

New Grants Drive DM Research

MDF and the Wyck Foundation jointly announce the funding of two new research projects. The projects address critical gaps in research infrastructure and clinical trial readiness and will increase understanding of the progression of DM, and provide measures to evaluate disease progression and the efficacy of candidate therapeutics.

3 Tips for Research Participants to Make Every Body Count

“Participating in a research study will help the greater good, and I’m hoping one may help me too,” says Caroline Easterling about why she participates in DM research. Read on for Caroline’s advice for participants.

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