WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim of the study is to specify meaningful outcome measures for future clinical trials. WiTNNess is open to children and adults worldwide. Participants can choose to include their information once (cross-sectional cohort) or every few months (prospective cohort).
TNNT1-associated Myopathy, Infantile-onset Nemaline Rod Myopathy, Myopathies, Nemaline, Myopathy, Myopathy, Rod, Myopathy; Hereditary, Amish Nemaline Myopathy, Nemaline Myopathy 5, NEM5, Genetic Muscle Disease, Recessive Hereditary Disorder (Autosomal), ANM
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim of the study is to specify meaningful outcome measures for future clinical trials. WiTNNess is open to children and adults worldwide. Participants can choose to include their information once (cross-sectional cohort) or every few months (prospective cohort).
WiTNNess - TNNT1 Myopathy Natural History Study
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Clinic for Special Children, Strasburg, Pennsylvania, United States, 17579
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
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Clinic for Special Children,
Kevin Strauss, MD, PRINCIPAL_INVESTIGATOR, Clinic for Special Children
2027-06-01