# Charcot–Marie–Tooth Association: Accelerating Research\. Empowering Patients\. > CMTA supports the development of new treatments for CMT, improvements in the quality of life for people with CMT, and efforts to find a cure\. Generated by Yoast SEO v28.7, this is an llms.txt file, meant for consumption by LLMs. ## Pages - [About CMTA](https://cmtausa.org/about-cmta/) - [Contact Us](https://cmtausa.org/contact-us/) - [Terms \& Conditions](https://cmtausa.org/terms-conditions/) - [Privacy Policy](https://cmtausa.org/privacy-policy/) - [CMTA Gadget Store](https://cmtausa.org/cmta-gadget-store/) ## Events - [Central Virginia Branch Meeting with Guest Speaker](https://cmtausa.org/events/central-virginia-branch-meeting-with-guest-speaker/) - [Minneapolis, MN CMTA Branch Social \(In\-person\)](https://cmtausa.org/events/minneapolis-mn-cmta-branch-social-in-person/) - [October Emotional Support Group Meeting](https://cmtausa.org/events/october-emotional-support-group-meeting/) - [Southern California Joint Branch Meeting with Ken Cornell](https://cmtausa.org/events/southern-california-joint-branch-meeting-with-ken-cornell/) - [Seattle, WA CMTA Branch meeting with Guest Speaker](https://cmtausa.org/events/seattle-wa-cmta-branch-meeting-with-guest-speaker/) ## Press Releases - [CMTA\-INC Alliance Announces 2027 Inherited Neuropathies Fellowship Recipient: Johns Hopkins’ Clinician\-Scientist Dr\. Brendan Zotter](https://cmtausa.org/press-release/cmta-inc-2027-fellowship/): CMTA\-INC Alliance awards its 2027 Inherited Neuropathies Fellowship to Johns Hopkins physician\-scientist Brendan Zotter, MD, PhD, who will develop improved AAV vectors for targeted gene therapy delivery in CMT\. - [CMTA Honors World\-Renowned Clinician Michael E\. Shy, MD, with Lifetime Achievement Award](https://cmtausa.org/press-release/shy-lifetime-achievement-award/): The Charcot\-Marie\-Tooth Association presented neurologist Michael E\. Shy, MD, with its first\-ever Lifetime Achievement Award, recognizing three decades of pioneering CMT research, patient care, and building the clinical trial infrastructure now advancing treatments\. - [CMTA Invests $110,500 in Novel ASO Medicine for CMT2A](https://cmtausa.org/press-release/aso-cmt2a-la-jolla-labs/): The Charcot\-Marie\-Tooth Association announced a $110,500 investment partnering with La Jolla Labs to develop an antisense oligonucleotide medicine targeting CMT2A, the most common axonal form of CMT, using a novel approach to increase MFN1 expression\. - [Prof\. Charlotte Sumner Joins CMTA Scientific Advisory Board](https://cmtausa.org/press-release/sumner-scientific-advisory-board/): CMTA announced the appointment of Charlotte Sumner, MD, to its Scientific Advisory Board, adding clinical and research expertise\. - [CMTA Earns a Perfect Rating From Charity Navigator](https://cmtausa.org/press-release/cmta-earns-perfect-charity-navigator-rating/): CMTA has earned a perfect score from Charity Navigator, receiving a four\-star rating for financial health, accountability, and transparency\. The recognition places CMTA among a small group of health\-related nonprofits meeting Charity Navigator’s highest standards for responsible stewardship\. ## Simply CMT - [10,000 Patients, One Goal: The First Treatments for CMT](https://cmtausa.org/simply-cmt/10000-patients-one-goal-the-first-treatments-for-cmt/) - [Anni Albers: Pushing Boundaries in Art and in Life ](https://cmtausa.org/simply-cmt/anni-albers-pushing-boundaries-in-art-and-in-life/): Anni Albers was an artist\. First a painter, then a weaver\. Then a printmaker, when her hand tremors made weaving too difficult\. - [A Backup Gene Offers a New Path to CMT2A Treatment ](https://cmtausa.org/simply-cmt/a-backup-gene-offers-a-new-path-to-cmt2a-treatment/): What if the answer to CMT2A is already in your cells? A new CMTA partnership is testing whether switching on a backup gene could protect nerves when MFN2 fails\. - [FDA to Hold Patient Listening Session for CMT2C/TRPV4 ](https://cmtausa.org/simply-cmt/fda-to-hold-patient-listening-session-for-cmt2c-trpv4/): This October, the FDA will host a Patient Listening Session for the CMT2C/TRPV4 community, organized by CMTA\. During this event, the FDA will hear from people living with this form of CMT about their lives, their needs, and their hopes for new treatments\. - [Your Braces Should Be Earning Their Keep: What a New Study Says About AFOs and CMT ](https://cmtausa.org/simply-cmt/new-study-afos-and-cmt/): In a new study, Dutch researchers found that AFOs can measurably improve how people with CMT walk, but it depends on how the braces are chosen and fitted\. ## Videos - [Bracing for CMT: Real Stories \& Expert Insights](https://cmtausa.org/video/bracing-for-cmt-real-stories-expert-insights-2026-q3-lunch-and-learn/) - [Surgery: CMT Surgical Outcomes \| 2024 CMTA Summit](https://cmtausa.org/video/surgery-cmt-surgical-outcomes-2024-cmta-patient-research-summit/) - [Stretch Break with Stone and Barnett \| 2024 CMTA Summit](https://cmtausa.org/video/stretch-break-1-with-julie-stone-and-julie-barnett-2024-cmta-patient-research-summit/) - [STAR Breakout: Type 4/GAN/SORD \| 2023 CMTA Summit](https://cmtausa.org/video/star-breakout-session-type-4-gan-sord-2023-patient-research-summit/) - [STAR Breakout: Type 2/Unknown \| 2023 CMTA Summit](https://cmtausa.org/video/star-breakout-session-type-2-unknown-2023-patient-research-summit/) ## Centers of Excellence - [CMTA Center of Excellence at Washington University School of Medicine in St\. Louis](https://cmtausa.org/centers-of-excellence/washington-university-school-of-medicine-st-louis/) - [CMTA Center of Excellence at Virginia Commonwealth University \(VCU\)](https://cmtausa.org/centers-of-excellence/virginia-commonwealth-university-coe/) - [CMTA Center of Excellence at UVA Health](https://cmtausa.org/centers-of-excellence/cmta-center-of-excellence-at-university-of-virginia-health/) - [CMTA Center of Excellence at UT Southwestern Medical Center](https://cmtausa.org/centers-of-excellence/cmta-center-of-excellence-at-university-of-texas-southwestern-medical-center/) - [CMTA Center of Excellence at UPMC Department of Neurology](https://cmtausa.org/centers-of-excellence/university-of-pittsburgh-medical-center-upmc-department-of-neurology/) ## Podcast Episodes - [CMTA CEO Sue Bruhn, PhD, on Leading Global CMT Research \& Patient Care](https://cmtausa.org/podcast-episodes/cmta-ceo-sue-bruhn-phd-on-leading-global-cmt-research-patient-care/) ## Research - [CMT2A Biomarkers and Natural History](https://cmtausa.org/research-projects/cmt2a-biomarkers-and-natural-history/): With CMTA support of $572,055, researchers at the Inherited Neuropathy Consortium led by Michael E\. Shy, MD, are working to track disease progression and identify biomarkers in CMT2A\. By studying blood, skin biopsy, and MRI biomarkers alongside clinical assessments, this project aims to develop critical tools for future clinical trials and advance CMT2A research\. - [Developing an MFN1\-Targeted ASO Therapy for CMT2A](https://cmtausa.org/research-projects/developing-mfn1-aso-cmt2a-treatment/): CMTA is funding researchers at La Jolla Labs, working with John Svaren, PhD, to develop an ASO treatment that increases MFN1 expression as a potential way to compensate for the damaging effects of MFN2 mutations that cause CMT2A\. - [Allele\-Specific Silencing of MFN2 to Treat CMT2A](https://cmtausa.org/research-projects/allele-specific-crispr-cmt2a/): What if one gene therapy could work for 200\+ CMT2A mutations? Dr\. Bruce Conklin's team is using CRISPR to silence the faulty MFN2 gene while leaving the healthy copy untouched\. - [Measuring Calf and Thigh Muscle Fat in CMT4J](https://cmtausa.org/research-projects/measurement-of-muscle-fat-fraction-in-calf-and-thigh-muscle-in-patients-with-cmt4j/) - [Long Read Sequencing in Undiagnosed Axonal CMT Patients](https://cmtausa.org/research-projects/long-read/): The answer is hiding in your DNA\. For some, it hasn’t been found yet\. This CMTA\-funded study uses long\-read sequencing to uncover genetic causes of CMT that standard tests may miss\. ## CMTA Branches - [State College](https://cmtausa.org/branches/state-college/) - [Lancaster Pennsylvania](https://cmtausa.org/branches/lancaster-pennsylvania/) - [Arizona](https://cmtausa.org/branches/arizona/) - [Utah](https://cmtausa.org/branches/utah/) - [Bucks County, PA](https://cmtausa.org/branches/bucks-county-pa/) ## CMT Clinicians - [Mark Treihaft, MD](https://cmtausa.org/clinicians/mark-treihaft-md/) - [Jahannaz Dastgir, DO](https://cmtausa.org/clinicians/jahannaz-dastgir-do/) - [Stephen F\. Conti, MD](https://cmtausa.org/clinicians/stephen-f-conti-md/) - [Joseph T\. Hogan, DPM](https://cmtausa.org/clinicians/joseph-t-hogan-dpm/) - [Vera Fridman, MD](https://cmtausa.org/clinicians/vera-fridman-md/) ## Profiles - [Lainie Ishbia](https://cmtausa.org/profiles/lainie-ishbia/) - [Sarah Krukar \| She/Her/Hers](https://cmtausa.org/profiles/sarah-krukar-she-her-hers/) - [Emmily Stufflet \| She/Her/Hers](https://cmtausa.org/profiles/emmily-stufflet/) - [Patricia Verduin, PhD](https://cmtausa.org/profiles/patricia-verduin-phd/): Pat Verduin brings decades of science and technology leadership and a personal connection to CMT1A to the CMTA Board of Directors\. - [Anatomic Inc\.](https://cmtausa.org/profiles/anatomic-inc/) ## Patients as Partners - [Genetic Counseling, Life Outlook and Family Planning in CMT](https://cmtausa.org/patients-as-partners/the-effects-of-genetic-counseling-on-life-outlook-attitudes-and-family-planning-in-cmt-patients/) - [Study of Intrathecal ELP\-02 for CMT4J](https://cmtausa.org/patients-as-partners/study-of-intrathecal-elp-02-for-charcot-marie-tooth-disease-type-4j-cmt4j/) - [CMT Subtype Biomarkers Study: Types 2A, 2F, 1B and X1](https://cmtausa.org/patients-as-partners/research-project-cmt-subtype-biomarkers-and-outcome-measures-study-for-cmt-types-2a-2f-1b-and-x/) - [OpenCap Markerless Motion Capture Study \(In\-Person Only\)](https://cmtausa.org/patients-as-partners/opencap-video-based-markerless-motion-capture-for-kinematic-analysis-in-person-only/) - [Gait, Mobility and Balance in CMT With Cavovarus Foot](https://cmtausa.org/patients-as-partners/lower-extremity-gait-mobility-and-balance-confidence-in-charcot-marie-tooth-patients-with-cavovarus-foot-deformity/) ## Publications - [CMT Respiratory Impairment](https://cmtausa.org/publications/cmt-respiratory-impairment/) - [CMT Guide for School Nurses](https://cmtausa.org/publications/cmt-guide-for-school-nurses/) - [2023 CMTA\-STAR Achievements](https://cmtausa.org/publications/2023-cmta-star-achievements/) - [An Introductory To SORD Deficiency](https://cmtausa.org/publications/an-introductory-to-sord-deficiency/) - [The CMTA \- Who We Are](https://cmtausa.org/publications/the-cmta-who-we-are/) ## Categories - [News](https://cmtausa.org/category/news/) - [Research](https://cmtausa.org/category/research/) - [Community](https://cmtausa.org/category/community/) - [CMT Awareness](https://cmtausa.org/category/cmt-awareness/) - [Genetic Therapy](https://cmtausa.org/category/research/genetic-therapy/) ## Center of Excellence Categories - [USA](https://cmtausa.org/centers-of-excellence/coe-categories/usa/) - [North America](https://cmtausa.org/centers-of-excellence/coe-categories/north-america/) - [Adult](https://cmtausa.org/centers-of-excellence/coe-categories/adult/) - [Pediatric](https://cmtausa.org/centers-of-excellence/coe-categories/pediatric/) - [California](https://cmtausa.org/centers-of-excellence/coe-categories/california/) ## Research Categories - [CMT1A](https://cmtausa.org/research-projects/research-categories/cmt1a/) - [CMT2 Research](https://cmtausa.org/research-projects/research-categories/cmt2/) - [CMT1 Research](https://cmtausa.org/research-projects/research-categories/cmt1/) - [Genetic Therapy](https://cmtausa.org/research-projects/research-categories/genetic-therapy/) - [CMT2A](https://cmtausa.org/research-projects/research-categories/cmt2a/) ## Clinician Categories - [Neurology](https://cmtausa.org/clinicians/clinician-categories/neurology/) - [All CMT Clinicians](https://cmtausa.org/clinicians/clinician-categories/all-cmt-clinicians/) ## Patient as Partner Categories - [CMT1A](https://cmtausa.org/patients-as-partners/pap-categories/cmt1a/) - [CMT2](https://cmtausa.org/patients-as-partners/pap-categories/cmt2/) - [Natural History and Biomarkers](https://cmtausa.org/patients-as-partners/pap-categories/natural-history-and-biomarkers/) - [Patient Perspective](https://cmtausa.org/patients-as-partners/pap-categories/patient-perspective/) - [CMT1](https://cmtausa.org/patients-as-partners/pap-categories/cmt1/) ## Research Subtypes - [CMT1](https://cmtausa.org/research-subtype/cmt1/) - [CMT1A](https://cmtausa.org/research-subtype/cmt1a/) - [CMT2](https://cmtausa.org/research-subtype/cmt2/) - [CMT1B](https://cmtausa.org/research-subtype/cmt1b/) - [CMT4](https://cmtausa.org/research-subtype/cmt4/) ## Therapy Approaches - [Small Molecule](https://cmtausa.org/therapy-approach/small-molecule/) - [Biomarkers and Natural History Study](https://cmtausa.org/therapy-approach/biomarkers-and-natural-history-study/) - [Genetic Therapy \- AAV](https://cmtausa.org/therapy-approach/genetic-therapy-aav/) - [Delivery](https://cmtausa.org/therapy-approach/delivery/) - [In Vitro Model](https://cmtausa.org/therapy-approach/in-vitro-model/) ## Video Category - [Patient and Research Summit](https://cmtausa.org/video-category/patient-and-research-summit/) - [Lunch and Learn](https://cmtausa.org/video-category/lunch-and-learn/) - [Webinar](https://cmtausa.org/video-category/webinar/) ## Optional - [Sitemap index](https://cmtausa.org/sitemap_index.xml)