New Perspectives on Respiratory Involvement in DM1

Respiratory dysfunction in DM1 draws new attention.

Decline in Daily and Social Activities in DM1

Daily activities and social participation are assessed in a longitudinal study of DM1.

New Research Study on Cognitive Function and Neuroimaging CDM

Dr. Melissa Dixon at the University of Utah Department of Pediatrics is conducting a study to learn more about how congenital myotonic dystrophy (CDM) and childhood-onset myotonic dystrophy affect thinking, memory, attention, brain function, and how these processes change over time.

Characterizing Pediatric DM1

A large natural history study of children with DM1 may form the background for subsequent development of evidence-based case management guidance.

Health-Related Quality of Life in DM1

A new review article synthesizes published data on health-related quality of life in adult-onset DM1.

Publication of Christopher Project Report

DM patient and family member/caretaker assessments of the burden of disease form the basis of a recent publication.

Cognitive and Adaptive Development in Congenital/Childhood DM1

A new longitudinal study focuses on CNS function in congenital and childhood DM1.

Lessons Learned: Targeting RNA as a Therapeutic Strategy

A new review article addresses advances in exploiting the biology of RNA with small molecule drug strategies.

A Novel Genome Editing Strategy for DM1

A novel genome editing strategy removes expanded CUG repeats in DMPK transcripts.

Drug Development Informed with TACT

TREAT-NMD’s TACT process provides unbiased feedback on drug development programs in neuromuscular diseases.

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