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.

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...

DTx Pharma Acquired by Novartis for CMT1A Therapy

Jul 17, 2023 | CMTRF Funded Research, Drug Development, Research News

Novartis acquires DTx’s FALCON technology to advance treatment development for CMT1A

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...

Dr. William W. Motley to Join CMT Research Foundation Board of Directors

Jun 21, 2023 | Press Releases

ATLANTA (June 21, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease*, today announced that Dr. William W. Motley, a neurologist with a doctorate in neurogenetics with fifteen...

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...

DTx Pharma Video – Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A

May 7, 2023 | CMTRF Funded Research, Drug Development, Research News

Thomas F. Sander Joins CMT Research Foundation Board of Directors

Apr 27, 2023 | Press Releases

ATLANTA (April 26, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced that rare disease sales specialist, Thomas F. Sander, has joined its Board of Directors....

Edritz Javelosa to Join the CMT Research Foundation as Chief Scientific Officer

Apr 17, 2023 | Press Releases

ATLANTA (April 17, 2023) Edritz Javelosa (Hav-a-low-sa), PhD, who has been at the Muscular Dystrophy Association (MDA) since 2019 and Research Portfolio Director since 2021, has joined the CMT Research Foundation (CMTRF) as Chief Scientific Officer. Reporting to...