Charcot-Marie-Tooth disease and the Possibilities with a New Treatment in a Clinical Trial

Living with Charcot-Marie-Tooth Disease

Living with Charcot-Marie-Tooth (CMT) disease means navigating a progressive nerve disorder that affects much more than movement. From early signs like frequent tripping, toe-walking, and trouble with fine motor skills, those living with CMT may experience symptoms such as:

  • Hand/arm weakness (reduced grip strength)

  • Difficulty with mobility (walking, stair climbing)

  • Balance

  • Ankle weakness (foot drop)

  • Muscle fatigue

  • Difficulty breathing

CMT can make everyday tasks feel exhausting. Despite its life-altering impact, CMT remains widely misunderstood and without an FDA-approved treatment. At our company, we appreciate the full reality of living with CMT - and we’re working to develop therapies that truly make a difference.

NMD Pharma’s Treatment Approach: Targeting the Skeletal Muscles in CMT

Currently there is no approved treatment for Charcot-Marie-Tooth disease.

NMD670 is an experimental oral medicine being tested to help people with CMT. It is a skeletal muscle-targeted therapy that is being investigated for the potential to increase muscle responsiveness to weak nerve signals and enable more consistent muscle function. It is an oral medication taken with a simple glass of water.

Join the Clinical Trial to Study the Potential Benefits and Safety of a New Investigational Skeletal Muscle-Targeted Drug

The SYNAPSE-CMT Study is evaluating an investigational drug called NMD670 in adults with Charcot-Marie-Tooth Disease in clinical sites located in the US, Europe and Canada.

The main purpose of this study is to learn how the study drug (NMD670) works and how safe the study drug is compared with placebo.

You may be able to take part in the SYNAPSE-CMT Trial if you are:

  • 18 to 70 years of age

  • Diagnosed with any genetically confirmed CMT 1 or CMT 2 subtype (note that this current study does not include patients with CMT 1X)

  • Able to perform certain physical tests without the use of an ankle/foot brace (orthopedic inserts are allowed)

  • Can attend 6 different in-clinic visits over a period of approximately 8 weeks (depending on scheduling)

This is not a complete list of study requirements. The study doctor will review the full requirements for this study with you. It is important to note that future studies are being considered which will include expanded eligibility criteria specific to wider age ranges, use of an ankle/foot brace and additional subtypes.

There is no cost to participate in this study, and your reasonable travel costs will be reimbursed (flight, mileage, hotel, parking, meals).

For more information and to request involvement in this important Charcot-Marie-Tooth disease clinical trial, including cities/states of clinical trial sites and contact information for clinical coordinators, please visit clinicaltrials.gov or email NMD Pharma at: clinicaltrials@nmdpharma.com

NMD670 has not been approved by Regulatory Health Authorities and the safety and effectiveness of NMD670 in SMA, MG or CMT has not been established