DM1

MDF SAC Member Profile: Dr. Kathie Bishop

MDF is pleased to welcome Dr. Kathie Bishop, Ph.D., to its Scientific Advisory Committee(SAC). Dr. Bishop, who joined the SAC in summer 2015, is a seasoned expert in neurological and neuromuscular research and drug development.

Benefit/Risk Study Results: Focus on Muscle Weakness

The Myotonic Dystrophy Foundation partnered with Silicon Valley Research Group to develop a survey to better understand how people with DM weigh the benefits of new treatments again the risks. 

Caregiving Today: Stresses, Satisfactions, and a Need for Better Solutions

In honor of National Family Caregivers Month, and to celebrate the many critically important caregivers in the MDF community, we’ve taken a look at the status of caregiving today. 

Evolving Strategies Using Oligonucleotides for DM1

Conjugation of morpholino phosphorodiamidate oligomer (PMO) ASOs to a cell-penetrating peptide improves their bioavailability and efficacy in skeletal muscle of a DM1 model.

Possible Muscle Damage Pathway Identified in DM

Researchers at the University of Virginia recently published a paper describing a biological pathway they believe may be responsible for muscle degeneration in DM1.

Myotonic Dystrophy Stem Cells

Researchers at the University of Florida, led by Dr. Tetsuo Ashizawa, recently published a study in which they developed a strategy for DM1 stem cell therapy involving gene modification.

Healthy Heart Information for American Heart Month

February is American Heart Month, and MDF has partnered with Drs. Katharine Hagerman and Marianne Goodwin to highlight important research on cardiac issues in myotonic dystrophy for our readers

Dr. Tetsuo Ashizawa's Multi-Disciplinary Approach

Dr. Tetsuo Ashizawa, MD, has focused his career on the search for DM treatments and care for those affected. His multi-disciplinary, patient-centric approach to care moves DM research out of the lab and into his clinic at the University of Florida.

Endocrine Function Over Time in Patients with Myotonic Dystrophy Type 1

Dr. John Vissing and his colleagues at the University of Copenhagen recently tracked a group of 68 adults with myotonic dystrophy type 1 (DM1), measuring their endocrine function change over 8 years.

Parental Age Effects in the Transmission of DM1

New research on the impact of the mother's age on congenitally affected offspring.

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