CMT1A is caused by the duplication of the Peripheral Myelin Protein 22 (PMP22) gene, which leads to the demyelination of the peripheral nerves. CMT1A patients have overexpression (too much) PMP22 protein.
HNF Research Initiatives
FDA Repurposed Drugs
In September 2021, HNF partnered with Rarebase, a public benefit company that leverages cutting-edge technology (Function, a tech-enabled drug discovery platform) and biology using FDA-approved drugs to deliver accelerated, off-label treatments for CMT1A. HNF has completed Phase I of the project.
Novel Disease Modifying Drugs
Pharnext Pharmaceuticals
A clinical-stage biopharmaceutical company, partnered with HNF in 2012 to support the development of PXT3003 for CMT1A. Currently Pharnext is expecting the results of the PREMIER Phase III clinical trial later this year.
Miralinc Pharmaceuticals
An early stage biotech was founded with the initial investment from HNF. Company has pioneered next generation Histone deacetylase subtype 6 inhibitors (HDAC6i’) to advance life-changing disease cures for several types of CMT, including CMT1A.
Natural History Studies
The Global Registry for Inherited Neuropathies (GRIN)
Developed in 2013 to stratify large groups of patients for all types of CMT to gain a deeper understanding of CMT. This important data also allows HNF to better identify the burden, diagnostic journey, and prevalence of CMT that will aid TRIAD research partners. Patient data = Improved Research & Clinical Trial Design.
CMT&Me App
A smartphone digital study sponsored by Pharnext Pharmaceuticals in collaboration with HNF, other CMT advocacy groups and experts in six countries to collect real-world anonymised data from adults (≥18 years) living with CMT. The aggregated data is available in close to real time, via dashboards and is analyzed based on pre-set criteria (ie. diseases stage, daily symptoms, age, demographics) to understand the real-world impact of CMT and the unmet needs of patients.
If you don’t know your CMT type, you can find out from the HNF through The CMT Genie Program.
www.cmtgenie.orgLearn more on this topic
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