Research

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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