Therapeutic area

Charcot-Marie-Tooth disease

Developing digital measures of gait, balance, daily activity, and upper limb function for research in children and adults with Charcot-Marie-Tooth disease.

Charcot-Marie-Tooth (CMT) disease is a group of genetic disorders that affect peripheral nerves controlling movement and sensation. CMT can cause progressive weakness, muscle wasting, sensory loss, foot drop, and balance difficulties, particularly in the feet, lower legs, and hands. Symptoms and progression vary by subtype and individual.

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LEGSys accepted into the FDA ISTAND program for CMT1A trials

LEGSys has been accepted into the FDA ISTAND program as a drug development tool for wearable sensor-derived measurement of foot drop in CMT1A.

See FDA’s CDER statement (opens in a new tab) View the qualification pathway

Our research in CMT

Our CMT research focuses on two complementary areas: evaluating wearable measures of function across CMT subtypes and age groups, and building datasets that complement clinical assessments and support future trials.

  • Wearable monitoring

    Digital measures for assessing CMT

    Evaluating gait, balance, activity, and upper limb measures against clinical assessments, with validation tailored to each population and intended use.

  • Research datasets

    Data to complement clinical trials

    Measurements of movement and daily activity that add context to performance assessed during clinic visits.

Digital measures for assessing CMT

We are developing wearable measures to study how CMT affects movement and daily activity in children and adults. Gait and balance assessments in the clinic are complemented by activity and upper limb monitoring at home. Research evaluates how these measures relate to clinical function across CMT subtypes.

  • LEGSys

    Wearable sensors that quantify gait and, with the appropriate sensor configuration, ankle movement and measures related to foot drop.

  • BalanSens

    Wearable sensors that estimate postural sway during standardized balance tests to support quantitative assessment of stability.

  • PAMSys

    Wearable sensors that measure steps, walking, posture, and activity patterns during daily life.

  • PAMSys ULM

    Wrist-worn sensors that record movement during daily activities, with algorithms estimating patterns of goal-directed upper limb use.

Research evidence for digital measures in CMT

Conference reports describe associations between wearable measures and clinical assessments in adults with CMT, as well as the feasibility of home monitoring. These cross-sectional findings support further research; sensitivity to disease progression or treatment-related change requires longitudinal evaluation.

  • Disease severity

    Assessment of disease severity

    A 2025 AAN abstract reported a study of 26 adults with CMT. Daily step counts, cadence, and selected balance measures were associated with scores on the CMT Functional Outcome Measure.

  • Remote monitoring

    At-home remote monitoring

    An MDA abstract reported 14 days of home monitoring in 26 adults with CMT. Activity and selected upper limb movement features were associated with clinical measures; goal-directed movement counts alone were not associated with overall disease severity.

FDA qualification pathway for our digital measures in CMT

The FDA's Innovative Science and Technology Approaches for New Drugs (ISTAND) program supports qualification of novel drug development tools for a defined context of use. The FDA has accepted our Letter of Intent, Wearable Sensor-Derived Foot Drop Metrics as Longitudinal Outcome Measures in Adult CMT1A Patients, into the FDA ISTAND program (DDT-IST-000073). The proposed context of use is longitudinal assessment in adults with CMT1A, the most common CMT subtype. Qualification remains in progress.

  • Letter of Intent

    Wearable foot drop metrics for CMT1A trials

    The submission proposes LEGSys-derived measures of ankle movement and foot drop, including ankle range of motion, foot drop angle, and mid-swing foot angle, for longitudinal assessment in adult CMT1A trials.

  • Patient priority

    Foot drop, a patient-prioritized symptom

    Foot drop can impair walking, increase the risk of tripping and falling, and limit independence. At the 2018 externally led Patient-Focused Drug Development meeting convened by the Hereditary Neuropathy Foundation, people with CMT highlighted its impact on daily life. These patient perspectives help guide the development of meaningful outcome measures.

  • Qualification process

    Advancing toward qualification

    Letter of Intent acceptance is an early step in FDA qualification. The process also includes a Qualification Plan and Full Qualification Package. Our work with the Hereditary Neuropathy Foundation and the CMT community aims to build the evidence needed for the proposed use.

Wearable measures in CMT drug trials

PAMSys and LEGSys support activity and gait assessment in CMT drug development. The randomized phase 2 ACE-083 study included wearable assessments, illustrating how digital measures can complement other trial outcomes.

Explore our publications

Our clinical study in CMT

Our work with the Hereditary Neuropathy Foundation evaluates gait, balance, daily activity, and upper limb measures in CMT. The summary below reflects BioSensics-reported study details. Published conference analyses describe a 26-adult cohort with 14 days of home monitoring.

  • Completed

    BioDigit CMT

    Reported enrollment
    48
    Reported follow-up
    Up to 2 years

    For sponsors developing endpoints in CMT, see Endpoint Development

Our research partners

Our CMT research is conducted in collaboration with academic medical centers, patient advocacy organizations, and industry partners.

  • Alesta Therapeutics
  • Elpida Therapeutics
  • Hereditary Neuropathy Foundation
  • Muscular Dystrophy Association
  • St. Jude Children's Research Hospital
  • Stanford University
  • University of Kansas Medical Center
  • University of Sydney

Selected publications and presentations

Advance CMT research with digital measures

Plan digital assessments for your CMT study. Discuss research collaborations, study protocols, and data access with our team.

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