At CMTA, we’re committed to improving the lives of those living with Charcot-Marie-Tooth (CMT) disease and other rare disorders. We proudly support impactful advocacy efforts to address the unique challenges faced by the CMT and rare disease communities. One key initiative we’re supporting is the Rare Pediatric Disease Priority Review Voucher (PRV) Program. Since its inception in 2012, the program has resulted in more than 50 new molecules for nearly 40 life-threatening rare pediatric disorders. However, this program is at risk of lapsing on December 20, which would be a significant setback for the rare disease community. We stand with the broader rare disease community in urging timely action to extend this program and ensure its life-changing impact continues. #CharcotMarieTooth #CMTDisease #LetsBeatCMT #CMTAFamily #Advocacy
Charcot-Marie-Tooth Association
非营利组织管理
Glenolden，Pennsylvania 2,717 位关注者
Our vision... a world without CMT!
关于我们
Our mission … to support the development of new treatments for CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure. Our vision … a world without CMT. About CMT: Named after the three doctors who first described it in 1886: Charcot, Marie, and Tooth, CMT affects one in every 2,500 people—a rare disease subdivided into multiple subtypes, each with a lower prevalence. Due to the degradation of their nerves, people with CMT suffer lifelong progressive muscle weakness and atrophy of the arms and legs, and can affect other parts of the body. This leads to problems with balance, walking, hand use, and more. There currently is no treatment or cure for this debilitating disease. About CMTA: The CMTA is a community-led, community-driven 501(c)(3) nonprofit organization with a mission to support the development of new treatments for CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure. As the leading global philanthropic funder of CMT research, the CMTA unites the community with clinicians and industry experts to accelerate the advancement of treatments, with investments of more than $24 million since 2008. For more information, visit the CMTA's website.
- 网站
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https://www.cmtausa.org
Charcot-Marie-Tooth Association的外部链接
- 所属行业
- 非营利组织管理
- 规模
- 11-50 人
- 总部
- Glenolden，Pennsylvania
- 类型
- 非营利机构
- 创立
- 1983
地点
Charcot-Marie-Tooth Association员工
动态
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Imagine a world where CMT is not just treatable but curable. With your help—and thanks to a matching gift opportunity—we are making strides toward that future every day. Led by the world’s foremost researchers and clinicians, the CMTA’s Strategy to Accelerate Research (CMTA-STAR) has more than 40 STAR alliance partners. Our integrated team of top scientists and industry partners are working on a host of research projects encompassing everything from gene therapy to small molecule therapies to drugs that promote axon survival and preserve nerve function. By making your year-end donation today, your gift will be matched to double its impact, helping fund life-changing research through the CMTA-STAR program and providing critical resources for those living with CMT. Make a gift today: https://lnkd.in/eHuhijCc #CMTResearch #CMTASTAR #CharcotMarieTooth #CMTDisease #LetsBeatCMT #CMTAFamily
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The Charcot-Marie-Tooth Association (CMTA) congratulates CMTA-STAR Alliance Partner NMD Pharma A/S on initiating its Phase II SYNAPSE-CMT trial. This significant milestone reflects a shared commitment to advancing therapeutic options for individuals living with CMT. The trial’s first patient was dosed under the direction of YESSAR HUSSAIN MD, PA DBA AUSTIN NEUROMUSCULAR CENTER, CMTA Centers of Excellence clinic director at the Austin Neuromuscular Center in Austin, Texas. Learn more at https://lnkd.in/gBnzkBah #CMTResearch #CMTASTAR #CharcotMarieTooth #CMTDisease #LetsBeatCMT #CMTAFamily
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The Charcot-Marie-Tooth Association (CMTA), through its strategic alliance with the Inherited Neuropathy Consortium (INC), is proud to announce the 2025 Inherited Neuropathy Fellowship. This one-year fellowship highlights how the CMTA-INC alliance drives progress by equipping the next generation of clinicians and researchers with the tools to advance clinical care and research in CMT. Fellowships will begin between February and August 2025, reinforcing the alliance’s mission to accelerate impactful advancements in CMT. Learn more and apply: https://lnkd.in/eTP4Uf5e #CMTResearch #CMTASTAR #CharcotMarieTooth #CMTDiseas #LetsBeatCMT #CMTAFamily
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Do you have CMT2C patients? Please share this with them! CMT2C / TRPV4 community members are invited to a meeting with Actio Biosciences, Inc. and the research team working on the CMT2C Natural History Study. Please register now and join us for this special meeting on Thursday, December 5th from 2:00 p.m. – 3:30 p.m. Eastern Time via ZOOM. You will hear updates on the study and upcoming clinical trials. Learn more and register: https://lnkd.in/eFW8QKTc #CMTAFamily #CMTAwareness #CMTResearch #CMTASTAR #CharcotMarieTooth #LetsBeatCMT
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What I’ve learned about finding an exercise regimen with CMT “In the current era of clinical trials, and with new treatments for CMT on the horizon, it’s more important than ever for people with CMT to be in the best possible shape, to keep as strong and mobile as we can, while we wait for approved treatments to become available.” - Dr. Katherine Forsey, CMTA's Chief Research Officer Full Story: https://lnkd.in/e9ZKH6e2 #CMTAFamily #LetsBeatCMT #CMTA #CMT #CharcotMarieTooth
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Fourth Quarter CMTA Lunch & Learn: Fireside Chat with Dr. Michael Shy Join us on December 4th when Dr. Michael Shy provides an exciting update on how CMTA’s partnership with the Inherited Neuropathy Consortium (INC) will play a key role in the future of CMT drug development. Also, CMTA’s Dr. Sue Bruhn and Dr. Katherine Forsey will discuss how CMTA is accelerating the pathway to new treatments for all types of CMT. Register Today: https://lnkd.in/edzbjayB #LetsBeatCMT #CMTAFamily #CMT #CMTA #CharcorMarieTooth
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Do you have CMT2C patients? Please share this with them! CMT2C / TRPV4 community members are invited to a meeting with Actio Biosciences, Inc. and the research team working on the CMT2C Natural History Study. Please register now and join us for this special meeting on Thursday, December 5th from 2:00 p.m. – 3:30 p.m. Eastern Time via ZOOM. You will hear updates on the study and upcoming clinical trials. Learn more and register: https://lnkd.in/eFW8QKTc #CMTAFamily #CMTAwareness #CMTResearch #CMTASTAR #CharcotMarieTooth #LetsBeatCMT
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CMTA and the Inherited Neuropathy Consortium (INC) – a global network of over 20 clinical research centers – have joined forces in a new strategic alliance that positions CMTA at the forefront of CMT research and patient care. Full Story: https://lnkd.in/eAvHZwwD #CMTAFamily #LetsBeatCMT #CharcotMarieTooth #CMT #CMTA
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Join us on Wednesday, December 4th at 12:00 p.m. Eastern Time for our 4th Quarter Lunch & Learn – a Fireside Chat with CMTA’s Dr. Sue Bruhn and Dr. Katherine Forsey. Drs. Bruhn and Forsey will discuss how CMTA is accelerating the pathway to new treatments for all types of CMT. Also, Dr. Michael Shy will be providing an exciting update on how CMTA’s partnership with the Inherited Neuropathy Consortium (INC) will play a key role in the future of drug development. Register now: https://lnkd.in/edzbjayB #CMT #CMTAwareness #CharcotMarieTooth #CMTAFamily #CMTStrong #LetsBeatCMT
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