# 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 v28.0, 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 - [Virginia Branch Meeting \(Virtual\) with Victoria Be](https://cmtausa.org/events/virginia-branch-meeting-virtual-with-victoria-be/) - [Texas CMTA Branches Meeting](https://cmtausa.org/events/texas-cmta-branches-meeting/) - [St\. Louis, MO CMTA Branch Meeting](https://cmtausa.org/events/st-louis-mo-cmta-branch-meeting-2/) - [Western Canada Branch Meeting \(Virtual\)](https://cmtausa.org/events/western-canada-branch-meeting-virtual/) - [Virginia CMTA Branch Meeting](https://cmtausa.org/events/virginia-cmta-branch-meeting-2/) ## Press Releases - [CMTA Appoints Aimee Yrlas Simpson as Chief Revenue Officer, Expanding Its Impact for People Living with CMT](https://cmtausa.org/press-release/cmta-appoints-aimee-yrlas-simpson/): CMTA appoints Aimee Yrlas Simpson as Chief Revenue Officer, bringing more than 25 years of nonprofit leadership experience to help advance research and support people living with CMT\. - [Geneticist Behind the First Identified Cause of CMT Joins CMTA\-STAR Scientific Advisory Board](https://cmtausa.org/press-release/vincent-timmerman-cmta-star/): Vincent Timmerman, PhD, the Belgian geneticist whose lab identified the first known genetic cause of Charcot\-Marie\-Tooth disease, joins the CMTA\-STAR Scientific Advisory Board\. Timmerman brings three decades of CMT genetics research and iPSC disease modeling expertise to CMTA's push to advance therapies into clinical trials\. - [CMTA Invests $141,000 to Study Hearing and Balance to Develop Better Clinical Trial Outcome Measures in Charcot\-Marie\-Tooth Disease](https://cmtausa.org/press-release/hearing-balance-study-cmt/): CMTA is supporting a two\-year research project examining hearing and balance signaling to strengthen outcome measures in future CMT clinical trials\. - [CMTA Receives Highly Competitive $154,000 American Camp Association Grant for Its Camp Footprint Program](https://cmtausa.org/press-release/camp-footprint-aca-character-camp-grant/): CMTA has received a $154,000 American Camp Association Character at Camp Implementation Grant to launch the Footprint Fellowship: Bringing Camp Magic Home\. The two\-year initiative extends the Camp Footprint experience beyond summer for 35 inaugural Fellows and positions the program as a national model for character\-driven camp programming\. - [CMTA Appoints David Stark, MD, and Catherine Illingworth to Board of Directors, Expanding Clinical and Biotech Expertise](https://cmtausa.org/press-release/cmta-appoints-david-stark-and-catherine-illingworth-to-board-of-directors/): CMTA welcomes David Stark, MD, and Catherine Illingworth to its Board of Directors, strengthening clinical insight and gene therapy leadership within the CMT community\. ## Simply CMT - [CMTA\-STAR Alliance Partner Vanda Pharmaceuticals Receives FDA Rare Pediatric Disease Designation for CMT2S Therapy Candidate ](https://cmtausa.org/simply-cmt/vanda-pharmaceuticals-fda-rare-pediatric-disease-designation-cmt2s/) - [More Miles, Less Fatigue: Walking Poles and CMT ](https://cmtausa.org/simply-cmt/walking-poles-and-cmt/) - [Is Spinal Fluid the Way to Gene Therapy in CMT? What a Decade of Research Reveals ](https://cmtausa.org/simply-cmt/spinal-fluid-and-gene-therapy/) - [CMTA Leading the Way in CMT1A](https://cmtausa.org/simply-cmt/leading-cmt1a-research/): CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments\. - [CMTA research takes the stage at PNS](https://cmtausa.org/simply-cmt/cmta-pns2026/): PNS 2026 showcased CMTA's role in advancing CMT research across the translational pipeline, from biomarkers and natural history studies to clinical trials, therapeutic development, and the scientific collaborations driving progress\. ## Videos - [Parenting Through CMT From Diagnosis to Independence: 2026 Q2 Lunch and Learn](https://cmtausa.org/video/parenting-through-cmt-from-diagnosis-to-independence-2026-q2-lunch-and-learn/) - [Genetic Testing \& Family Planning: 2026 Q1 Lunch and Learn](https://cmtausa.org/video/genetic-testing-family-planning-2026-q1-lunch-and-learn/) - [CMTA\-STAR Leading the Way](https://cmtausa.org/video/cmta-star-research-leading-the-way/) - [Ink \& Impact: CMT Stories in Print 2](https://cmtausa.org/video/ink-impact-cmt-stories-in-print-2/) - [Ink \& Impact: CMT Stories in Print](https://cmtausa.org/video/ink-impact-cmt-stories-in-print/) ## Centers of Excellence - [CMTA Center of Excellence at the Medical University of South Carolina \(MUSC\)](https://cmtausa.org/centers-of-excellence/cmta-center-of-excellence-at-the-medical-university-of-south-carolina-musc/) - [CMTA Center of Excellence at the University of Utah](https://cmtausa.org/centers-of-excellence/the-university-of-utah/) - [CMTA Center of Excellence at Chi Health Clinic Neurology Institute \(Immanuel\)](https://cmtausa.org/centers-of-excellence/chi-health-clinic-neurology-institute-immanuel/) - [CMTA Center of Excellence at Yale School of Medicine](https://cmtausa.org/centers-of-excellence/yale-school-of-medicine/) - [CMTA Center of Excellence at Children's Health of Dallas Specialty Center at the University of Texas Southwest](https://cmtausa.org/centers-of-excellence/childrens-health-of-dallas-speciality-center-at-the-university-of-texas-southwest/) ## 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 - [Blood–Brain Barrier\-Crossing Conjugates for Treating CMT](https://cmtausa.org/research-projects/cmt-gene-therapy-rna-delivery/): CMTA\-funded research at Mount Sinai is studying how RNA medicines can be delivered into nerve cells in CMT, overcoming biological barriers that block treatment\. - [Preliminary Characterization of STEP Platform for Delivery of Genome Editing Therapy for CMT](https://cmtausa.org/research-projects/step-delivery-cmt/): CMTA\-funded Yale researchers are testing STEP, a non\-viral delivery system for genome\-editing medicines in CMT\. - [Applied Therapeutics \- INSPIRE](https://cmtausa.org/research-projects/applied-therapeutics-inspire/) - [Development of a Human\-Derived Myelin\-Containing Organoid as a Reference Model for CMT1A](https://cmtausa.org/research-projects/cmt1a-organoid/): Researchers led by CMTA\-STAR Advisory Board member Vincent Timmerman, PhD, at the University of Antwerp are developing human\-derived CMT1A organoids using patient\-derived stem cells\. This research platform aims to improve understanding of disease mechanisms and provide a standardized model for evaluating potential treatments\. - [Schwann Cell\-Targeted Peptide\-LNPs for Delivery of siRNA Against PMP22 in CMT1A](https://cmtausa.org/research-projects/peptide-lnp-cmt1a/) ## CMTA Branches - [Research Triangle Area, NC](https://cmtausa.org/branches/raleigh-research-triangle-park-nc/) - [Wilmington, NC](https://cmtausa.org/branches/wilmington-nc/) - [Los Angeles, CA](https://cmtausa.org/branches/los-angeles/) - [Kansas City, KS](https://cmtausa.org/branches/kansas-city-ks/) - [Jacksonville, FL](https://cmtausa.org/branches/jacksonville-fl/) ## 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 - [Augustine Therapeutics](https://cmtausa.org/profiles/augustine-therapeutics/) - [Calico Life Sciences LLC](https://cmtausa.org/profiles/calico-life-sciences-llc/) - [Aimee Yrlas Simpson](https://cmtausa.org/profiles/aimee-yrlas-simpson/) - [Lainie Ishbia](https://cmtausa.org/profiles/lainie-ishbia/) - [Herbert Beron](https://cmtausa.org/profiles/herbert-beron-secretary/) ## Patients as Partners - [The Effects of Genetic Counseling on Life Outlook Attitudes and Family Planning in CMT Patients](https://cmtausa.org/patients-as-partners/the-effects-of-genetic-counseling-on-life-outlook-attitudes-and-family-planning-in-cmt-patients/) - [GeneTIC Patient Perspectives Study](https://cmtausa.org/patients-as-partners/genetic-patient-perspectives-study/) - [Development of CMTPedS Scale for Children with CMT: INC Research Study 6603](https://cmtausa.org/patients-as-partners/inc-6603-cmtpeds-scale/) - [Charcot\-Marie\-Tooth disease Infant Scale: INC Research Study 6611](https://cmtausa.org/patients-as-partners/inc-6611-cmt-infant-scale/) - [Your Participation Matters: Join the CMT2S Research Study](https://cmtausa.org/patients-as-partners/your-participation-matters-join-the-cmt2s-research-study/) ## 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/) - [Biomarkers and Natural History](https://cmtausa.org/research-projects/research-categories/biomarkers-and-natural-history/) ## 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/) - [CMT1](https://cmtausa.org/patients-as-partners/pap-categories/cmt1/) - [Patient Perspective](https://cmtausa.org/patients-as-partners/pap-categories/patient-perspective/) - [All Research Opportunities](https://cmtausa.org/patients-as-partners/pap-categories/all-research-opportunities/) ## 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/) - [Genetic Therapy \- CRISPR](https://cmtausa.org/therapy-approach/genetic-therapy-crispr/) ## 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)