The RYR-1 Foundation
Strength In Numbers
The RYR-1 Foundation
EIN: 47-1094057
as of November 2023
as of November 13, 2023
Mission
The RYR-1 Foundation's mission is to support research leading to an effective treatment or a cure for RYR-1-related diseases by: 1) Research: We make grants to researchers interested in RYR-1-related diseases and developing a patient registry. 2) Medical Professional Education: The vast majority of medical professionals have never heard of RYR-1-related diseases. We raise awareness through resources on our website, as well as direct meetings with medical professionals around the world. 3) Patient/Family Support and Advocacy: We serve as a resource for patients and their families through our website, other forms of social media, and biennial International Family Conferences.
Ruling year
2017
President & Co-Chair of Research
Dr. Michael Falk Goldberg M.D.
Vice President & Co-Chair of Research
Dr. Morton Falk Goldberg
Main address
P.O. Box 13312
Pittsburgh, PA 15243 USA
Formerly known as
RYR1 Foundation
The RYR1 Foundation
EIN
47-1094057
Diseases and conditions
Genetic conditions and birth defects
Community and economic development
Population served
Adults
People with diseases and illnesses
NTEE code
Birth Defects, Genetic Diseases Research (H20)
Diseases of Specific Organs (G40)
Alliance/Advocacy Organizations (S01)
IRS subsection
501(c)(3) Public Charity
IRS filing requirement
This organization is required to file an IRS Form 990 or 990-EZ.
Tax forms
Communication
Programs and results
What we aim to solve
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Patient Registry
There are several reasons why this project is so critical. This database will serve to:
1) Increase our understanding of RYR-1 muscle disease: RYR-1 gene related abnormalities have been associated not only in congenital myopathy, but also malignant hyperthermia, intolerance of certain cholesterol lowering drugs ("statin myopathy"), and exercise intolerance. Building a patient database will allow physicians, researchers, and genetic counselors to better understand the different forms of RYR-1 myopathy, its natural history, and its prevalance.
2) Conduct clinical trials: As a rare disease, RYR-1-related diseases can present a challenge to researchers, pharmaceutical companies, and biotech firms who want to test potential therapies. Without easy access to patients who can serve as study subjects, clinical trials cannot be performed. Thus, by developing a database of RYR-1 patients, we can collaborate with those who have found potentially promising therapies.
Research Grants
The RYR-1 Foundation exists to raise funds that support both basic science and clinical research of RYR-1-related diseases. Funding research gives us the best chance to find a treatment or a cure for this disease.
As of 2021, The RYR-1 Foundation has funded/committed over $1.7 million in RYR-1-related research. This has already lead to important advances in knowledge and potential therapies.
RYR-1 International Family Conference
The RYR-1 Foundation held the first ever RYR-1 International Family Conference in July 2016 in Baltimore and the second in July 2018 in Pittsburgh. Combined, these events have hosted 388 attendees, representing 75 RYR-1-affected families from 31 states and eight countries. These conferences are unique opportunities for affected individuals and families to meet, form friendships, gain knowledge, and develop a true RYR-1 community.
For a rare condition like RYR-1-related diseases, providing a forum for affected individuals and families to meet others like them has significant psychological value. It is a goal of The RYR-1 Foundation to enhance and enrich these relationships within the RYR-1 community by hosting biennial International Family Conferences.
The third RYR-1 International Family Conference is scheduled for July 22-24, 2022 in Pittsburgh, PA USA.
To view a documentary about the RYR-1 International Family Conferences, please go to: www.ryr1.org/conferences.
Clinical Care Guidelines for RYR-1-Related Diseases: What Patients and Families Need to Know
The RYR-1 Foundation was awarded a $50,000 grant from the Oscar & Elsa Mayer Family Foundation to produce a handbook for patients and families affected by RYR-1-related diseases. Difficulty with accessing reliable and easily comprehended information on these rare conditions is a tremendous source of frustration and anxiety for affected individuals and families. The RYR-1 Foundation was able to produce an informative guide with practical and easily understood information. This handbook is free to the public and available for download in multiple languages: www.ryr1.org/ccg.
Chapters Included:
1. The Genetics of RYR-1-RD
2. Clinical Features of RYR-1-RD
3. The Role of Calcium and the Ryanodine Receptor in RYR-1-RD
4. Malignant Hyperthermia (MH)
5. The Lungs/Pulmonology
6. The Bones and Joints/Orthopedics
7. Eating, Swallowing, & Speaking
8. Considerations for School
9. Physical Activity & Physical Therapy (PT)
10. Is There a Treatment for RYR-1-RD?
RYR-1 Research Meetings
International research meetings are essential for the dissemination of new scientific discoveries. They are also important for networking and the sharing of ideas among clinicians, basic science researchers, and the pharmaceutical industry. The RYR-1 Foundation has sponsored various meetings, including: Gordon Research Conference, World Muscle Society, International Congress on Neuromuscular Diseases, and European Muscle Conference. The Foundation has also co-hosted and sponsored a meeting at the European Neuromuscular Centre specifically on RYR-1-related diseases.
The RYR-1 Foundation hosts biannual Scientific Advisory Board (SAB) meetings.
Funding Basic & Clinical Research
Acquiring funding for clinical research for a rare disease can be challenging for clinicians and scientists. As a small organization focusing on a single condition, The RYR-1 Foundation has worked successfully with researchers to obtain funding for the translation of basic research to future clinical trials.
Patient-Led International RYR-1 Scientific Workshop
The RYR-1 Foundation will host it's first ever Patient-Led International RYR-1 Scientific Workshop on July 20-22, 2022 in Pittsburgh, PA USA. This workshop will consist of affected individuals, researchers, doctors, and medical professionals.
Where we work
Affiliations & memberships
NORD 2016
External reviews
Videos
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 identify and remedy poor client service experiences, To identify bright spots and enhance positive service experiences, 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
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Which of the following feedback practices does your organization routinely carry out?
We take steps to ensure people feel comfortable being honest with us, We look for patterns in feedback based on demographics (e.g., race, age, gender, etc.)
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What challenges does the organization face when collecting feedback?
We don't have any major challenges to collecting feedback
Financials
Financial documents
Download audited financialsRevenue vs. expenses: breakdown
Liquidity in 2021 info
240.37
Months of cash in 2021 info
49.1
Fringe rate in 2021 info
13%
Funding sources info
Assets & liabilities info
Financial data
The RYR-1 Foundation
Balance sheetFiscal Year: Jan 01 - Dec 31
The balance sheet gives a snapshot of the financial health of an organization at a particular point in time. An organization's total assets should generally exceed its total liabilities, or it cannot survive long, but the types of assets and liabilities must also be considered. For instance, an organization's current assets (cash, receivables, securities, etc.) should be sufficient to cover its current liabilities (payables, deferred revenue, current year loan, and note payments). Otherwise, the organization may face solvency problems. On the other hand, an organization whose cash and equivalents greatly exceed its current liabilities might not be putting its money to best use.
Fiscal Year: Jan 01 - Dec 31
This snapshot of The RYR-1 Foundation’s financial trends applies Nonprofit Finance Fund® analysis to data hosted by GuideStar. While it highlights the data that matter most, remember that context is key – numbers only tell part of any story.
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Business model indicators
| Profitability info | 2017 | 2018 | 2019 | 2020 | 2021 |
|---|---|---|---|---|---|
| Unrestricted surplus (deficit) before depreciation | $674,799 | $36,173 | $75,935 | $163,384 | $34,914 |
| As % of expenses | 175.6% | 8.6% | 21.8% | 49.5% | 13.5% |
| Unrestricted surplus (deficit) after depreciation | $674,799 | $36,173 | $75,935 | $163,384 | $34,914 |
| As % of expenses | 175.6% | 8.6% | 21.8% | 49.5% | 13.5% |
| Revenue composition info | |||||
|---|---|---|---|---|---|
| Total revenue (unrestricted & restricted) | $1,059,068 | $778,647 | $744,214 | $393,570 | $519,685 |
| Total revenue, % change over prior year | 248.2% | -26.5% | -4.4% | -47.1% | 32.0% |
| Program services revenue | 0.4% | 4.2% | 4.9% | 21.7% | 29.3% |
| Membership dues | 0.0% | 0.0% | 0.0% | 0.0% | 0.0% |
| Investment income | 0.2% | 0.8% | 3.3% | 3.2% | 0.4% |
| Government grants | 0.0% | 0.0% | 0.0% | 0.0% | 2.7% |
| All other grants and contributions | 99.1% | 94.8% | 84.6% | 72.6% | 65.8% |
| Other revenue | 0.2% | 0.2% | 7.2% | 2.6% | 1.7% |
| Expense composition info | |||||
|---|---|---|---|---|---|
| Total expenses before depreciation | $384,269 | $421,185 | $349,004 | $330,396 | $257,682 |
| Total expenses, % change over prior year | 282.4% | 9.6% | -17.1% | -5.3% | -22.0% |
| Personnel | 11.4% | 12.3% | 17.6% | 17.7% | 22.3% |
| Professional fees | 4.8% | 8.1% | 11.6% | 11.2% | 16.8% |
| Occupancy | 0.0% | 0.0% | 0.0% | 0.0% | 0.0% |
| Interest | 0.0% | 0.0% | 0.0% | 0.0% | 0.0% |
| Pass-through | 70.8% | 41.0% | 51.8% | 55.3% | 44.7% |
| All other expenses | 13.1% | 38.6% | 18.9% | 15.8% | 16.2% |
| Full cost components (estimated) info | 2017 | 2018 | 2019 | 2020 | 2021 |
|---|---|---|---|---|---|
| Total expenses (after depreciation) | $384,269 | $421,185 | $349,004 | $330,396 | $257,682 |
| One month of savings | $32,022 | $35,099 | $29,084 | $27,533 | $21,474 |
| Debt principal payment | $0 | $0 | $0 | $0 | $0 |
| Fixed asset additions | $0 | $0 | $0 | $0 | $0 |
| Total full costs (estimated) | $416,291 | $456,284 | $378,088 | $357,929 | $279,156 |
Capital structure indicators
| Liquidity info | 2017 | 2018 | 2019 | 2020 | 2021 |
|---|---|---|---|---|---|
| Months of cash | 28.7 | 33.7 | 51.5 | 34.0 | 49.1 |
| Months of cash and investments | 37.0 | 43.1 | 63.5 | 70.4 | 98.8 |
| Months of estimated liquid unrestricted net assets | 35.3 | 33.2 | 42.7 | 51.0 | 67.0 |
| Balance sheet composition info | 2017 | 2018 | 2019 | 2020 | 2021 |
|---|---|---|---|---|---|
| Cash | $917,695 | $1,181,163 | $1,498,917 | $935,646 | $1,054,536 |
| Investments | $267,393 | $331,993 | $349,030 | $1,002,860 | $1,066,033 |
| Receivables | $50,000 | $0 | $50,000 | $13,000 | $116,290 |
| Gross land, buildings, equipment (LBE) | $0 | $0 | $0 | $0 | $0 |
| Accumulated depreciation (as a % of LBE) | 0.0% | 0.0% | 0.0% | 0.0% | 0.0% |
| Liabilities (as a % of assets) | 8.9% | 1.5% | 1.1% | 0.1% | 0.4% |
| Unrestricted net assets | $1,128,894 | $1,165,067 | $1,241,002 | $1,404,386 | $1,439,300 |
| Temporarily restricted net assets | $0 | $324,845 | N/A | N/A | N/A |
| Permanently restricted net assets | $0 | $0 | N/A | N/A | N/A |
| Total restricted net assets | $0 | $324,845 | $646,362 | $545,027 | $788,737 |
| Total net assets | $1,128,894 | $1,489,912 | $1,887,364 | $1,949,413 | $2,228,037 |
Key data checks
| Key data checks info | 2017 | 2018 | 2019 | 2020 | 2021 |
|---|---|---|---|---|---|
| Material data errors | No | No | No | No | No |
Operations
The people, governance practices, and partners that make the organization tick.
Documents
President & Co-Chair of Research
Dr. Michael Falk Goldberg M.D.
Mike is also a co-founder of The RYR-1 Foundation. He is a board-certified neuroradiologist and is the Director of Neuroradiology at the Allegheny Health Network. As someone who is affected by an RYR-1-related disease, he understands first-hand the importance of building a community of similarly-affected individuals and families. As a physician, he has used his medical and scientific background to lead The RYR-1 Foundation’s efforts to fund critically important RYR-1 research. In a short period of time, The RYR-1 Foundation’s research program has had a major international impact and has led to important scientific and medical breakthroughs.
Vice President & Co-Chair of Research
Morton Goldberg
Mort is also a co-founder of The RYR-1 Foundation. He is also an ophthalmologist specializing in retinal disease and genetics. He is the Joseph Green Professor of Ophthalmology at the Johns Hopkins School of Medicine and the Director Emeritus of the Wilmer Eye Institute. He is a member of the Institute of Medicine of the National Academies of Science of the United States.
Number of employees
Source: IRS Form 990
The RYR-1 Foundation
Officers, directors, trustees, and key employeesSOURCE: IRS Form 990
Compensation data
There are no highest paid employees recorded for this organization.
The RYR-1 Foundation
Board of directorsas of 11/05/2023
Board of directors data
Dr. Michael Goldberg
The RYR-1 Foundation
Term: 2021 - 2024
Dr. Morton Goldberg
The RYR-1 Foundation
Term: 2021 - 2024
Morton Goldberg
The RYR-1 Foundation
Jennifer Ryan
The RYR-1 Foundation
Michael Legum
The RYR-1 Foundation
Emily Pedersen
The RYR-1 Foundation
Randolph Pepper
The RYR-1 Foundation
Donald Zack
The RYR-1 Foundation
Justin McArthur
The RYR-1 Foundation
Michael Goldberg
The RYR-1 Foundation
Andrew Huseth
The RYR-1 Foundation
Robert Hanich
The RYR-1 Foundation
Bryan Heft
The RYR-1 Foundation
Myrna Goldberg
The RYR-1 Foundation
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? No -
CEO oversight
Has the board conducted a formal, written assessment of the chief executive within the past year ? No -
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? No -
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:
The organization's co-leader identifies as:
Race & ethnicity
Gender identity
Sexual orientation
Disability
We do not display disability information for organizations with fewer than 15 staff.
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