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2022 Annual Report_spreads
…development for many types of CMT, including CMT1A, CMT1X, CMT2A, CMT2D, CMT2E, CMT2F, CMT2S, CMT4A and CMT4C, utilizing a spectrum of different technologies, including AAV delivery, gene silencing, gene replacement,…
Applied Therapeutics Announces Positive Sorbitol Reduction Data From the Ongoing Phase 3 INSPIRE Trial in Sorbitol Dehydrogenase (SORD) Deficiency
…evaluating the effect of once-daily oral AT-007 in approximately 50 patients age 16-55 with SORD Deficiency in the US and Europe. SORD Deficiency (also called SORD Neuropathy or CMT-SORD) is…
Ask the Expert: Braces, Orthotics, and Footwear
…(pronated) and mobile type CMT foot. The foot orthotic often becomes ineffective as the CMT progresses and the foot deformity advances and worsens. This is when shoe modifications like rocker…
Clinical Trials
…Disease CMT Research Study: Evaluation of Changes on Gait in Children and Youth with CMT The CMT&ME digital study: How CMT affects patients’ daily lives Research Survey About Impacts of…
Awareness Month: A STAR Researcher’s Perspective
…ten years that we’ve been trying to develop gene therapy initially for the x-linked type of CMT and, more recently, also for CMT4C, CMT1A and CMT2A. We work very hard…
Sperm Donor with Gene for Charcot-Marie-Tooth Disease Passes It on to Multiple Families
…using Donor X’s sperm. Both have a rare form of CMT—Dominant Intermediate B, a group of CMT subtypes in which nerve conduction does not comport with either axonal CMT or…
The 2019 Fall CMTA Report
…We Are Family: A Message from CEO Amy Gray Stage Set for Type 2 Breakthroughs New Gene Therapy Development Program for CMT2A Announced CMTA Board Approves Extension of CMT2E Project…
Genetics-of-CMT
…CMT subtype Gene HNPP PMP22 (deletion) CMT1A PMP22 (duplication) CMT1B MPZ CMT1C LITAF/SIMPLE CMT1D EGR2 CMT1E PMP22 (point mutation) CMT1X GJB1 (Cx32) CMT2A MFN2 CMT2B RAB7 CMT2D GARS CMT2E NFL…
CMTA Funds Cutting-Edge Gene Editing Studies for Type 2 CMT
…use a $653,000 grant from the CMTA in 2020 to develop the gene-editing technique known as CRISPR for application to CMT2A, 2E and 2F. Gene editing is a group of…
New CEO Takes Helm of the CMTA
Published on December 13, 2018. Last edited by CMTA Admin on June 28, 2019. Amy J. Gray The CMTA Board of Directors announced May 15 the appointment of Amy J….