News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

Endgame Campaign for CMT1A Tops $6.6 Million

Sep 20, 2023 | CMT Research Updates, CMTRF Funded Research

Recently, Peter J. deSilva, CMTRF Board member and Chair of the ENDGAME Capital Campaign to end CMT1A and Susan Ruediger, CMT patient, co-founder, and Chief Mission Officer of the CMTRF, hosted a private call with donors to update them on the progress being made in...

Oryzon Develops Novel HDAC6 Inhibitor ORY-4001 Showing Therapeutic Potential for CMT in Mouse Model

Jul 18, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

Scientists have been studying a potential treatment option for CMT by inhibiting (blocking) a protein called histone deacetylase 6 (HDAC6). They have found that HDAC6 plays an important role in regulating the pathways involved in breaking down proteins and...

Joao Cardoso, Paralympic Table Tennis Player with CMT2A

Jul 11, 2023 | About CMT, Life with CMT

Meet Joao Cardoso, a paralympic table tennis player from Portugal, living in France, and competitor in this year’s International Para Championship. In his 5 years of competing, Joao has earned many awards and titles, including the Medal of Sporting Merit and this...

Taking on the Yorkshire Three Peaks Challenge for Charcot-Marie-Tooth (CMT)

Jun 1, 2023 | About CMT, Life with CMT

Two women in the UK, Lucy Brown and Chrysi Albustin, will be tackling the Yorkshire Three Peaks Challenge to raise awareness and funds for Charcot-Marie-Tooth (CMT). Lucy’s mother was diagnosed with CMT1A in 2002; before Lucy was born. CMT is a progressive,...

Mother and Three Sons, All Diagnosed with CMT2N within a Year

May 14, 2023 | About CMT, Life with CMT

Just one year ago, Tara’s oldest son, Luca, started coming home from school crying in pain. He would rush to take off his shoes and socks, saying that his feet were “buzzing”. At five years old, Luca’s foot had high arches and a deformity, causing his bones to...

DTx Pharma

May 8, 2023 | About CMT, News

We are pleased share this story that published on May 3: https://patientworthy.com/2023/05/03/dtx-pharma-developing-cmt1a-rna-therapeutics/ One of the early programs CMTRF funded was DTx, a young company that had developed a new platform for RNA therapeutics. DTx was...

A Rare Disease Throughout Time

Feb 28, 2023 | About CMT, Life with CMT

More than 3 million people around the world have Charcot-Marie-Tooth (CMT) disease, but even with its quirky name, CMT is still relatively unknown. A progressive, degenerative disease involving the peripheral nerves that branch out from the brain and spinal cord to...

CMT Research Foundation and Nanite Inc. Announce Partnership to Enhance Therapeutic Efficacy of Antisense Oligonucleotides in CMT1A

Feb 22, 2023 | CMT Research Updates, CMTRF Funded Research, Press Releases, Research News

ATLANTA, GA (February 22, 2023) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced a partnership with Nanite, Inc., a Boston-based biotechnology company developing...

Continuation of Inflammation Research for CMT1B

Feb 14, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

In 2020, the CMT Research Foundation had funded renowned nerve disease expert, Dr. Rudolf Martini of University Hospital Würzburg (Germany), to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients...

Optimal Dosing Identified in CMT1X Research Update

Jan 31, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

A CMT Research Foundation-funded project at the University of Illinois Chicago has identified optimal dosing for a potential therapeutic for CMT1X. Like other forms of CMT, cell-to-cell communication within the peripheral nervous system malfunctions in people with...