Carlo Besta Neurological Institute
Rome, Lazio 00144
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.
Cedars Sinai Medical Center
Los Angeles, California 90048
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Children's Hospital at Westmead
Westmead, New South Wales 2145
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Connecticut Children's Medical Center
Hartford, Connecticut 06106
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Great Ormond Street Hospital for Children
London, Greater London WC1N 3JH
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.
Hospital Sick Children Toronto
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Houston Methodist Research Institute
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.
Johns Hopkins University
Baltimore, Maryland 21218
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Massachusetts General Hospital
Boston, Massachusetts 02114
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
National Hospital for Neurology and Neurosurgery
London, Greater London WC1N 3BG
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Seattle Children's Hospital
Seattle, Washington 98105
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
St. Jude Children's Research Hospital
Memphis, Tennessee 38105
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
Stanford University
Stanford, California 94305
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
University of Colorado
Aurora, Colorado 80045
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
University of Iowa - Administrative Core
Iowa City, Iowa 52242
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.
University of Miami
Miami, Florida 33136
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
University of Michigan
Ann Arbor, Michigan 48109
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
University of Minnesota
Minneapolis, Minnesota 55455
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina 27514
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
University of Pennsylvania
Philadelphia, Pennsylvania 19104
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.
University of Rochester
Rochester, New York 14627
Recruiting
6601: Natural History Evaluation of Charcot Marie Tooth Disease Third-party Collaboration
A lack of high quality natural history data, based on a uniform, quantitative evaluation of patients continues to hinder the efforts to perform clinical trials for most forms of CMT. This study aims to determine the natural history of CMT1B, CMT2A, CMT4A, CMT4C, and other types of CMT in order to refine the overall picture of disease for use in future clinical trials.
6602: Genetics of CMT- Modifiers of CMT1A, New Causes of CMT Third-party Collaboration
Despite great success of gene identification in CMT, there is still much more genetic heterogeneity to uncover. In addition, modifying factors are an important aspect of CMT, but have been rarely studied in a systematic fashion. This study will apply innovative study designs and the latest technology to tackle some of the most pressing genetic issues in CMT, ultimately paving the way for new therapeutic approaches. This study aims to determine if gene modifiers exist for CMT1A, and to find new genetic causes of CMT.
6613: Digital Measures of Physical Activity, Gait and Balance in CMT Third-party Collaboration
Gait and balance deficits resulting from CMT are core contributors to disability. Thus, it is important to reliably and sensitively capture the impact of candidate therapies on daily activity, especially on gait and balance. This study aims to characterize real world function by measuring habitual physical activity of individuals with different Charcot-Marie-Tooth disease (CMT) subtypes and evaluate the natural history of physical activity over a 3 year period, and to validate digital biomarkers of gait and balance deficits in individuals with CMT by evaluating the reliability, sensitivity, and responsiveness of parameters derived from wearable inertial sensors.