Charcot-Marie-Tooth Association

aka CMTA   |   Glenolden, PA   |  www.cmtausa.org

Mission

Our mission … to support the development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure.

Ruling year info

1986

CEO

Mrs. Amy Gray

Main address

P.O. Box 105

Glenolden, PA 19036 USA

Show more contact info

EIN

22-2480896

NTEE code info

Fund Raising and/or Fund Distribution (G12)

Nerve, Muscle and Bone Diseases (G50)

IRS filing requirement

This organization is required to file an IRS Form 990 or 990-EZ.

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Communication

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Our mission … to support the development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure.

Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

Strategy to Accelerate Research

In 2008, the CMTA launched STAR as a strategic research program to maximize breakthroughs in genetics and dramatically speed up the pace of CMT research. The opportunities associated with STAR stem from the fact that the causes of CMT have been pinpointed, leading to the identification of more than 90 specific gene defects. The Strategy involves: the creation of cell lines/assays (tests). Our scientists replicate the CMT cells of mice/rats and place them in tiny dishes. Next, we robotically test hundreds of thousands of compounds (drugs) of interest on these cells to see how they react to potential medications. Parallel with the creation of cell-based assays and the high-throughput process, we create animal models with each disease type and test the most promising medications on them.

Population(s) Served
Adults

Where we work

Our results

SOURCE: Self-reported by organization

How does this organization measure their results? It's a hard question but an important one.

Number of paid participants in conferences

This metric is no longer tracked.
Totals By Year
Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

These conferences allow people with CMT and their families to get up-to-date information on research from the scientists. They also feature presentations from local clinicians & health care providers.

Number of Facebook followers

This metric is no longer tracked.
Totals By Year
Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

The purpose of our Facebook group is to discuss CMT-related matters, ask CMT-related questions, and to share our experiences with CMT in order to help and support one another.

Percent of expense dollars spent on Mission

This metric is no longer tracked.
Totals By Year
Related Program

Strategy to Accelerate Research

Type of Metric

Input - describing resources we use

Direction of Success

Increasing

Context Notes

Our mission … to support the development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure.

Number of campers enrolled

This metric is no longer tracked.
Totals By Year
Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

The CMTA's Camp Footprint is the only camp in the USA just for kids with CMT! It is an annual, FREE, five-day sleepaway camp for youth with CMT. It is about building confidence and friendships!

Our Sustainable Development Goals

SOURCE: Self-reported by organization

Learn more about Sustainable Development Goals.

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

The CMTA's overarching goal is to find a drug treatment for CMT. While it searches for a treatment, its goal is to provide support and education to the CMT community.

STAR IS BASED ON FIVE CORE BUSINESS PRINCIPLES:
1. Develop a strategy based on knowing the cause of the disease and where to focus.
2. Find the best researchers in the world and ask them to implement projects that support the strategy.
3. Create accountability—Hold those researchers accountable for achieving their goals. We take your money very seriously. Our researchers are not fully paid until they fully deliver.
4. Demand collaboration—We bring the researchers out of their silos to collaborate with each
other. We are now seeing more and more technologies and therapies emanating from many
different fields of study.
5. Encourage partnerships—It costs between $400 million and $1 billion to bring a new drug to
market. Like other nonprofits, the CMTA does not have this kind of money. We work with big pharmaceutical companies that have the money to develop drugs.

We created an advisory board with top-notch researchers. The Scientific Advisory Board has 14 world-class scientists. Because the work of STAR involves translational research—turning science into therapies—we created a Therapy Expert Board (TEB)–a group of experts who tell us how good the science is in terms of turning it into therapies for those with CMT. More recently, we realized we had to get ready for clinical trials so we created the Clinical Expert Board (CEB), a group of experts who are helping us and our partners think about how to design clinical trials.

For CMT1A, which accounts for approximately 90 percent of diagnosed cases, we've done animal studies, performed high-throughput screening, identified hits and worked with a company called Genzyme. Today, we're focused on two families of compounds that are being fine-tuned in the lab. Genzyme, a traditional pharmaceutical company, takes a small molecule approach, which utilizes chemical formulas. In parallel, we are also working with biotech companies, which create biological living proteins, or large molecules. For example, a company came to us with a very interesting approach—RNA interference—which inserts little pieces of DNA into nerves to affect the way the cells create the protein that overexpresses PMP22. We've seen promising results in rat testing. This technology is currently used in two approved drugs on the market.

In the decade since the CMTA’s research efforts were consolidated under the banner of STAR (Strategy to Accelerate Research), the STAR alliance has now expanded to more than 30 pharmaceutical, biotech and service partners. The CMTA has spent more than $16 million to finance scores of research projects with 50 of them currently active.

How we listen

SOURCE: Self-reported by organization

Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.

done We demonstrated a willingness to learn more by reviewing resources about feedback practice.
done We shared information about our current feedback practices.
  • Who are the people you serve with your mission?

    We serve the people who are impacted by Charcot-Marie-Tooth disease. This included the patient and their caregivers. We do this by funding promising research that will develop new drugs to treat and/or cure CMT disease. The CMTA also meets the needs of patients by providing resources such as a quarterly newsletter, (800) info line, literature about CMT, Patient-Family Conferences, Branches, and Centers of Excellence to help support and educate. To help support our younger community of CMT patients, we have started a Youth Council and Camp Footprint. Through participation in a community of youth with CMT, our young people make friendships based on shared experiences and understanding.

  • How is your organization collecting feedback from the people you serve?

    Focus groups or interviews (by phone or in person), Suggestions from the community,

  • How is your organization using feedback from the people you serve?

    To make fundamental changes to our programs and/or operations, To inform the development of new programs/projects, To strengthen relationships with the people we serve, To understand people's needs and how we can help them achieve their goals,

  • What significant change resulted from feedback?

    When the COVID pandemic hit, we changed all 'in person' meetings such as Branch meetings and our Patient Family Conference to virtual. We learned that our some in our community prefer virtual meetings based on the attendance numbers at these meetings and feedback from our Branch leaders. We have decided that with future 'in person' events, we will continue to offer a virtual component so all may participate.

  • With whom is the organization sharing feedback?

    The people we serve, Our staff, Our board, Our funders, Our community partners,

  • How has asking for feedback from the people you serve changed your relationship?

    The CMTA was founded as an organization to help people with Charcot-Marie-Tooth disease. By including the people we serve in our decision making over formats of meetings, educational material, different community events, etc, we are better able to serve them. We rely heavily upon volunteers from the community in all that we do. The CMTA is pleased to announce the launch of our Patients as Partners in Research initiative! The goal of Patients as Partners in Research is to advance the work of our Strategy to Accelerate Research (STAR) and enroll the patient community in the critical work of furthering the development of treatments and ultimately a cure for CMT. The CMTA would not be in existence if we did not have our community.

  • Which of the following feedback practices does your organization routinely carry out?

    We aim to collect feedback from as many people we serve as possible, We take steps to ensure people feel comfortable being honest with us, We look for patterns in feedback based on people’s interactions with us (e.g., site, frequency of service, etc.), We engage the people who provide feedback in looking for ways we can improve in response, We act on the feedback we receive, We tell the people who gave us feedback how we acted on their feedback, We ask the people who gave us feedback how well they think we responded,

  • What challenges does the organization face when collecting feedback?

    We don't have any major challenges to collecting feedback,

Financials

Charcot-Marie-Tooth Association
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Operations

The people, governance practices, and partners that make the organization tick.

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Connect with nonprofit leaders

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  • Analyze a variety of pre-calculated financial metrics
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  • Compare nonprofit financials to similar organizations

Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.

Charcot-Marie-Tooth Association

Board of directors
as of 12/8/2021
SOURCE: Self-reported by organization
Board chair

Mr. Gilles Bouchard

Livescribe

Term: 2016 - 2022

Herb Beron

Morgan Stanley

Gilles Bouchard

Livescribe

Thomas Dubensky

Aduro

Lara Fava

Gary Gasper

Ernst & Young LLP

Alan Korowitz

Premiere Networks

Steven O'Donnell

Retired

Christopher Ouellette

Alpha Analytical

Phyllis Sanders

The Sanders Law Firm

Steven Scherer

University of Pennsylvania

Michael Shy

University of Iowa

John Svaren

University of Wisconsin

Board leadership practices

SOURCE: Self-reported by organization

GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.

  • Board orientation and education
    Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes
  • CEO oversight
    Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes
  • Ethics and transparency
    Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes
  • Board composition
    Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes
  • Board performance
    Has the board conducted a formal, written self-assessment of its performance within the past three years? No

Organizational demographics

SOURCE: Self-reported; last updated 2/1/2021,

Who works and leads organizations that serve our diverse communities? GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.

Leadership

No data

Race & ethnicity

No data

Gender identity

No data

 

No data

Sexual orientation

No data

Disability

No data