Resources worth your time

A focused collection for understanding CMT4C, making care decisions with professionals, navigating school and daily life, tracking research, and finding people who understand.

Reviewed August 2026 · External links open in a new tab

Useful before overwhelming

We favor official government, academic, clinical, and established CMT organizations. We look for material that is current, transparent about its purpose, and genuinely useful to families.

This library is a starting point, not an endorsement of every statement on an outside site. Research listings are not treatment recommendations, and educational material cannot replace advice from Jack's—or anyone else's—care team.

01

Understanding CMT4C

Reliable explanations of the condition, the SH3TC2 gene, inheritance, diagnosis, and the wider CMT family.

02

Care, mobility & daily living

Practical guidance to help frame conversations about therapy, braces, movement, safety, and day-to-day independence.

03

School & accommodations

Starting points for explaining CMT to a school and understanding disability protections in U.S. public education.

04

Research, registries & trials

The most important current CMT4C treatment research, plus places to contribute de-identified experience and look for studies. Eligibility always comes from the study team—not a web page.

Charcot-Marie-Tooth AssociationLatest update

Project Foresee: February 2026 development update

The clearest current progress report: FDA pre-IND feedback is complete, with drug manufacturing and formal toxicology work planned as the next steps toward a first-in-human study.

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PubMed · Gene TherapyMay 2026 paper

2026 dose-escalation and safety study

The newest peer-reviewed CMT4C gene-replacement study reports dose-dependent functional and nerve improvements without observed tissue toxicity or immune reactions in the treated mouse model.

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Charcot-Marie-Tooth AssociationProject hub

Project Foresee overview and family campaign

The project’s history, scientific team, milestones, funding campaign, and goal of moving an AAV-based SH3TC2 gene therapy toward patients.

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PubMed · Molecular Therapy2023 paper

SH3TC2 gene replacement: foundational proof of concept

The 2023 study behind Project Foresee showed improvements after both early and later treatment in a CMT4C mouse model using AAV9 to deliver a working SH3TC2 gene to Schwann cells.

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PubMed · Brain Communications2024 paper

A second experimental path: NT-3 gene therapy

A separate 2024 preclinical study found functional, electrical, and tissue-level improvements after an AAV1 vector was used to produce neurotrophin-3 in a CMT4C mouse model.

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ClinicalTrials.gov · NIH

Search studies for Charcot-Marie-Tooth disease

The U.S. government registry for current, completed, and upcoming clinical studies. Check subtype and eligibility carefully.

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Hereditary Neuropathy Foundation

Global Registry for Inherited Neuropathies (GRIN)

A patient registry that gathers lived-experience and genetic information to support research and trial readiness.

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Inherited Neuropathy Consortium

CMT research studies and clinical sites

A research network focused on different forms of CMT, with study listings, clinical sites, and patient-and-family information.

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05

Community & finding help

Directories and support services that can help families locate knowledgeable clinicians and connect with the broader neuromuscular community.

Found something that belongs here?

As this community grows, we will keep reviewing the library, replace outdated links, and add resources that meet the same standard.

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