Current Research

There are researchers, scientists, academic centers, pharmaceutical companies, and advocacy organizations working today to improve the understanding of CMT4J for a more hopeful tomorrow. Because CMT4J is rare, much of the information currently available comes from the experiences shared by the individuals living with CMT4J and from molecular biology research on the variants in the FIG4 gene that lead to reduced function of the FIG4 enzyme and ultimately lead to CMT4J. Information about a CMT4J Natural History study is available.

A Natural History study is an important step to advance understanding of a disease

Information about a CMT4J Natural History study is available

A Natural History study for Charcot-Marie-Tooth Type 4J (CMT4J) is now enrolling patients. Click here.

For more information about the natural history study, please contact:

What is a natural history study?

Why is a CMT4J natural history study important?

The Commitment of Neurogene to CMT4J

In addition to sponsoring this educational website, Neurogene Inc. is a biotech company committed to developing life-changing medicines for patients and families affected by rare, devastating neurological diseases.

Neurogene is partnering with researchers and clinicians to gain a greater understanding of CMT4J in an effort to treat the disease effectively. More information about Neurogene’s research pipeline can be found here.

Living CMT4J |
Living CMT4J |

CMT4J Studies

A clinical study entails research involving humans with the intended goal of strengthening medical knowledge. There are two types of clinical studies: non-interventional and clinical trials. Natural history studies are non-interventional, which means that there are no therapeutic interventions. In clinical trials, participants help researchers evaluate the effectiveness, safety, and side effects of investigational therapies that correlate with the disease’s development that may have the possibility of treating a disease.

Studies in CMT4J are registered on ClinicalTrials.gov, a website that provides access to information on publicly and privately supported human clinical (treatment) studies and natural history (non-interventional) studies on global diseases and conditions.

Searching “CMT4J” in the search bar on ClinicalTrials.gov will result in a list of upcoming trials that are not yet recruiting, trials that are currently enrolling, and trials that have concluded, including any published data resulting from completed studies.

Living CMT4J |