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2012 - A Year of Progress for Everyone with CMT

Dear Supporter of the CMTA,

We are writing today to share with you the successes accomplished by the CMTA this year. As you know, we are diligently working toward tomorrow's treatments while providing the resources to improve the lives of those with CMT today. The CMTA is the place where scientific experts, the patient and caregiver community, and the world's best CMT clinicians come to connect.

In 2012, the CMTA has significantly expanded our resources to the CMT community. The growing Support and Action Group network has reached over 7,000 members. Our community has been able to connect with world-class clinicians who are a part of the CMTA's Advisory Board both in person through our Patient/ Family Conference in November, and virtually, through the CMTA's "Ask the Experts" discussion forum on our website.

Because of funding from the CMTA, the researchers committed to finding a treatment for CMT have reached major milestones. STAR is currently funding the following research projects:

  1. Two full time specialists at the National Institutes of Health in Washington, DC, focused on High Throughput Screening and assay development
  2. Dr. Chan, CalTech, focused on creating Type 2A cellular assays
  3. Dr. Zuchner, University of Miami, focused on creating a Type 2A rat model
  4. Dr. Svaren, University of Wisconsin, focused on Type 1 and Type 2 cellular assays
  5. Dr. Battacharrya, University of Wisconsin, focused on human IPS cellular assays
  6. Dr. Nave and Dr. Serada, the Max-Planck Institute, Germany, focused on testing compounds in vivo Type 1A
  7. Dr. Martini, University of Wurzburg, Germany, focused on stem cell pilot rat study, Type X
  8. Dr. Liem, Columbia University, focused on creating cellular assays for Type 2

These milestones in STAR give us all hope for the future. But STAR also uncovers new diagnostic science and related symptoms, thus offering new knowledge about CMT to patients today. Through STAR, we have identified commonalities in symptoms previously not acknowledged as being related to CMT. We have published a new Medication List and we are developing a natural history study to understand the variant symptoms of CMT, even within the same family.

Great strides have been made in public awareness with increasing participation and a potential Presidential proclamation. The CMTA's Facebook fans have grown from under 2,000 in May to over 6,000 by November 1. In a Chase Community Giving Campaign, the CMTA placed 163 out of 30,000 non-profits and is a recipient of a $10,000 award based on the number of votes obtained. Over 17,000 people visit our website monthly looking for answers about CMT which greatly aids in their understanding and acceptance of CMT.

Jacqueline

Jacqueline is an eight-year-old with CMT type 1D, and her symptoms are devastating. Her CMT has progressed to complete deterioration of the muscles in her feet, hands, legs, arms and trunk, preventing her from using them. Her lung capacity is severely compromised due to atrophy of her diaphragm. Her ability to swallow is quickly deteriorating, and her dependence upon her mother and father for daily activities increases weekly. Her grandmother and aunt also suffer from the symptoms of CMT.

Jacqueline's family has come to the CMTA to find peer support, updated diagnostic information, and resources to increase awareness in their community. On the CMTA’s website they have found many helpful tools: the informational brochures to help educate her physicians, information about developing an Individualized Education Plan (IEP) for Jacqueline, and access to the advisory board to answer ongoing questions and concerns. Through the CMTA Jacqueline and her extended family found their local Support and Action Group where they have made friends with others with CMT, found support, and participated in local awareness efforts. Because of the CMTA, Jacqueline and her family better understand CMT and the expected progression in each person. They also have hope for the future – hope that their family's legacy will come to an end in this generation and that no other families will have to endure the devastating effects of CMT.

As you can imagine, our programs are invaluable to Jacqueline and her family. Providing these programs and research takes significant funding. We need your continued help to make scientific progress and provide the continued support to the community. We are proud that all 11 members of the Board of Directors have committed to raise or contribute $50,000 each.

Additionally, in 2011, the Support and Action Groups nationwide raised over $160,000 collectively. Our community is feeling empowered to be a part of this excitement and growth. Clearly, we have the support of the leaders of the CMTA, but we cannot stop there.

As we end 2012, we have planned 2013 to be another year of significant progress, not only in research, but also in community support and awareness. Our programs will continue to deliver significant benefits to those with CMT: increased patient and caregiver meetings and conferences, increased public and community awareness validating the debilitating symptoms of CMT, and lastly, the hope that the first treatment is on the horizon.

In order for the CMTA to achieve these goals, we need your continued support. Please join the Board of Directors, the Support and Action Group Facilitators, and Jacqueline's family by giving to the CMTA. Your support will allow us to continue to work toward tomorrow’s treatment while providing the resources to improve the lives of those with CMT today.

 

We in the CMT community can all benefit from a gift to the CMTA, today and tomorrow. We appreciate your continued support and your engagement with our organization.

Most sincerely,

HerbSig

Herb Beron
Chairman of the Board of Directors

PatSig

Patrick Livney
Chief Executive Officer

 

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