THE NEUROMUSCULAR DISEASE FOUNDATION
Join the Global Effort to Cure Neuromuscular Disease
Programs and results
What we aim to solve
The Neuromuscular Disease Foundation (NDF) has provided close to $10M of funding to critical research toward a treatment to end GNEM. We are now paving the way to a treatment that we believe will halt the progression of GNEM in patients, by working with leaders in drug development and genetic therapy research. Every cent donated helps us get closer to the human dosing stage of this treatment to end GNEM as we know it.
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Clinical Research
Funding critical Research is a cornerstone of our mission to find treatments and a cure for NMD, specifically GNE Myopathy. We currently fund research that supports our efforts in Gene Therapy, such as Whole genomic sequencing, RNA, development of human cell lines, bio-banking, the pursuit of mouse models, and essential science to better understand the role of mutations and modifiers.
Advocacy & education
Our primary goal while we wait for a cure is to mobilize our communities to identify, educate and support our patient population by getting the word out. We host events, patients days, and seminars that bring patients, families, and caregivers together with scientists and physicians to better understand all possibilities.
Outreach and Awareness
With the help of our volunteer community in tandem with patients and other advocates, we strive to create awareness in two main ways: 1. through contact with primary Physicians and Neurologists to expedite proper diagnosis. And 2. Through genetic screenings with the Public to identify and educate carriers, in hopes of prevention through IVF options.
Where we work
External reviews
Photos
Videos
Our results
How does this organization measure their results? It's a hard question but an important one.
Number of research studies funded
This metric is no longer tracked.Totals By Year
Population(s) Served
People with diseases and illnesses
Related Program
Clinical Research
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Context Notes
NDF has continued to fund GNEM-related clinical research and therapies that expedite treatments and, ultimately, a cure. The majority of studies we fund directly support the gene therapy process.
Number of support groups offered
This metric is no longer tracked.Totals By Year
Population(s) Served
Adolescents, Adults, Caregivers
Related Program
Advocacy & education
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Context Notes
NDF offers support groups for patients of GNE Myopathy (HIBM), caregivers/family members of patients, for teenage children of people diagnosed with GNEM and for those interested in being screened.
Number of clients participating in educational programs
This metric is no longer tracked.Totals By Year
Population(s) Served
People with physical disabilities, Chronically ill people
Related Program
Outreach and Awareness
Type of Metric
Context - describing the issue we work on
Direction of Success
Increasing
Context Notes
Our 2020 GNEM Virtual Speaker Series was launched as a response to COVID and our inability to host our annual Symposia. We produced 40 hours of content from 22 different speakers over 18 weeks.
Number of organizations applying for grants
This metric is no longer tracked.Totals By Year
Related Program
Clinical Research
Type of Metric
Output - describing our activities and reach
Direction of Success
Holding steady
Context Notes
The number of organizations we supplied grants to for each year.
Goals & Strategy
Learn about the organization's key goals, strategies, capabilities, and progress.
Charting impact
Four powerful questions that require reflection about what really matters - results.
What is the organization aiming to accomplish?
Funding *critical research* in Whole Genome Sequencing (WGS) to bolster our understanding of the role of GNE as the gene responsible for the disease, as well as any other mutations and/or modifiers. Also funding *Gene Therapy* Trials and supporting the NIH phase 2b Trials via directed funds towards ManNAc; a potential Treatment.
Increasing awareness and knowledge of GNEM by working with International scientists (in 15 Countries) to share data via an NDF-led Consortium on GNE Myopathy. We promoting *genetic carrier-screenings* prior to having children in order to prevent passing down the gene unnecessarily. Finally, we aim to improve the quality of life for our patients through education, advocacy, and Certified Advocacy Training for International patients and caregivers.
What are the organization's key strategies for making this happen?
We host an Annual Symposium on GNE Myopathy bringing Patients, Families, and Scientists together in Los Angeles. We provide Travel Scholarships for Patients and caregivers to attend this each year and allocate funds towards our International Patient events - following our own, to be led by and for other patients. We select, train, support and empower existing patients to serve as Advocacy and Outreach Program Managers and certified Advocates in many U.S. cities, as well as in 15 Countries in Europe, Asia, and the Middle East. We are currently funding scientists as Principal Investigators, and require that they share data via NDF's Scientific Consortium on GNEM.
What are the organization's capabilities for doing this?
With support from our prestigious Scientific Advisory Board, highly engaged Board of Directors, professional staff, and dedicated (trained) volunteers we are able to identify key areas of research at the intersection of biology and technology; spread the news, and advocate for change and growth in a way that satisfies our goals.
What have they accomplished so far and what's next?
In the past three years alone, we have expanded beyond a handful of Patients in Los Angeles (believed to be of one ethnicity) to hundreds of culturally diverse patients globally. We have established Certified Patient Advocates in 12 Cities from LA and NY to Italy and Israel, and have hosted International events geared to sharing our collective data in a thoughtful and complete way. Next year, we are on track to host 4 others. We have funded the pre-IND process for Gene Therapy, and having shown sufficient "Proof of Concept" to the FDA, our scientists are ready for the next steps prior to phase 1.
How we listen
Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.
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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 identify where we are less inclusive or equitable across demographic groups, To strengthen relationships with the people we serve
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Which of the following feedback practices does your organization routinely carry out?
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What challenges does the organization face when collecting feedback?
We don't have any major challenges to collecting feedback
Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
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Connect with nonprofit leaders
SubscribeBuild relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.
- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- 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.
THE NEUROMUSCULAR DISEASE FOUNDATION
Board of directorsas of 03/29/2022
Ralph Loren
Tricia Mullins
Ralph Loren
Locke Lord
Carolyn Yashari Becher
HopSkipDrive
Tricia Mullins
Horizon Pharma
Tahseen Mozaffar
UC Irvine
David Haverim
Maria Montgomery
Jason Taban
David Dahl
Shawn Arianpour
Tara Voogel
Board leadership practices
GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.
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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? Yes
Organizational demographics
Who works and leads organizations that serve our diverse communities? Candid partnered with CHANGE Philanthropy on this demographic section.
Leadership
The organization's leader identifies as:
Race & ethnicity
Gender identity
Transgender Identity
Sexual orientation
No data
Disability
We do not display disability information for organizations with fewer than 15 staff.
Equity strategies
Last updated: 11/06/2019GuideStar partnered with Equity in the Center - an organization that works to shift mindsets, practices, and systems to increase racial equity - to create this section. Learn more
- We review compensation data across the organization (and by staff levels) to identify disparities by race.
- We analyze disaggregated data and root causes of race disparities that impact the organization's programs, portfolios, and the populations served.
- We disaggregate data to adjust programming goals to keep pace with changing needs of the communities we support.
- We employ non-traditional ways of gathering feedback on programs and trainings, which may include interviews, roundtables, and external reviews with/by community stakeholders.
- We disaggregate data by demographics, including race, in every policy and program measured.
- We have long-term strategic plans and measurable goals for creating a culture such that one’s race identity has no influence on how they fare within the organization.
- We have a promotion process that anticipates and mitigates implicit and explicit biases about people of color serving in leadership positions.
- We seek individuals from various race backgrounds for board and executive director/CEO positions within our organization.
- We have community representation at the board level, either on the board itself or through a community advisory board.
- We help senior leadership understand how to be inclusive leaders with learning approaches that emphasize reflection, iteration, and adaptability.
- We measure and then disaggregate job satisfaction and retention data by race, function, level, and/or team.
- We engage everyone, from the board to staff levels of the organization, in race equity work and ensure that individuals understand their roles in creating culture such that one’s race identity has no influence on how they fare within the organization.