Form 990 for tax year 2023

Totals

Total revenue

$13,881,085

Total expenses

$12,112,486

Contributions and grants received

$12,660,929

Grants paid

$2,560,127

Program service revenue

$1,210,969

Investment income

$88,569

Other revenue

-$79,382

Total assets, end of year

$10,440,900

Total liabilities, end of year

$634,715

Net assets or fund balances, end of year

$9,806,185

Program services expenses (Part IX)

$10,187,712

Management and general expenses

$1,372,227

Fundraising expenses

$552,547

Program service expenses (Part III)

$10,187,712

Program services were 84.1% of total expenses calculated — Part IX's program-services column over total expenses, both as filed.

Line items

Every line the filer filed in Parts VIII, IX and X, as filed and in filed order. Blank columns are left out, and the lines are not forced to add up to the totals above.

Mission and programs · 2023

• RESEARCHPPMD'S RESEARCH PROGRAM IDENTIFIES AND AGGRESSIVELY FUNDS THE MOST PROMISING NEAR AND LONG-TERM DUCHENNE RESEARCH ACROSS NUMEROUS THERAPEUTIC STRATEGIES; STIMULATES NEW RESEARCH TO ENSURE THAT THE THERAPEUTIC PIPELINE IS RICH WITH OPPORTUNITY; FACILITATES PRE-COMPETITIVE INDUSTRY INTERACTIONS THROUGH THE DUCHENNE DRUG DEVELOPMENT ROUNDTABLE TO ENHANCE AND HARMONIZE THE CLINICAL TRIAL LANDSCAPE; AND SUPPORTS MULTI-STAKEHOLDER DATA MODELING CONSORTIA TO ACCELERATE DRUG DEVELOPMENT TIMELINES. IN ADDITION TO SIGNIFICANT PRE-CLINICAL AND ACADEMIC RESEARCH FUNDING, THROUGH THE VENTURE PATHWAYS PROGRAM, PPMD MAKES INVESTMENTS IN EARLY-STAGE BIOPHARMACEUTICAL COMPANIES TO CATALYZE DEVELOPMENT OF NOVEL THERAPIES TO TREAT DUCHENNE AND BECKER. THE DUCHENNE REGISTRYTHE DUCHENNE REGISTRY IS A PATIENT-REPORTED REGISTRY FOR INDIVIDUALS WITH DUCHENNE AND BECKER MUSCULAR DYSTROPHY AND CARRIER FEMALES, ESTABLISHED BY PPMD IN 2007, THE REGISTRY WAS CREATED TO BRIDGE THE INFORMATION GAP BETWEEN CLINICIANS, RESEARCHERS, AND THE PATIENT COMMUNITY, THEREBY ADDRESSING MEDICAL CARE NEEDS AND ACCELERATING THE PACE OF THERAPEUTIC ADVANCEMENTS. OVER 5,000 FAMILIES ACROSS THE GLOBE HAVE EMBRACED THEIR ROLE AS CITIZEN SCIENTISTS AND HAVE CONTRIBUTED TO THE LARGEST PATIENT-REPORTED DATA SET FOR DUCHENNE.SINCE ITS LAUNCH, DATA HAS BEEN REFERENCED IN THIRTEEN PUBLICATIONS AND EXPORTED BY RESEARCHERS 65 TIMES. THE REGISTRY HAS ALSO BEEN USED TO IDENTIFY AND CONNECT PEOPLE WITH DUCHENNE TO OVER 80 ACTIVELY RECRUITING TRIALS. THE DUCHENNE OUTCOMES RESEARCH INTERCHANGE (THE INTERCHANGE): THE INTERCHANGE COLLECTS PATIENT DATA AND CLINICIAN ENTERED DATA SO BOTH SETS ARE SO BOTH AGGREGATED AND ANALYZED IN ONE PLACE AND KEY INDUSTRY STAKEHOLDERS CAN GET A FULL PICTURE OF HOW DIFFERENT PEOPLE LIVE WITH DUCHENNE. THE ELECTRONIC HEALTH RECORD (EHR) STUDY: THE EHR STUDY EXTRACTS DATA FROM SEVERAL OF PPMD'S CERTIFIED DUCHENNE CARE CENTERS ACROSS THE COUNTRY TO IMPROVE CARE AND ANSWER RESEARCH QUESTIONS IN A WAY THAT DOES NOT REQUIRE EXTRA EFFORT FROM PATIENTS OR THEIR DOCTOR.THE REGISTRY IS ALSO HOME TO PPMD'S ELECTRONIC HEALTH RECORD (EHR) EXTRACTION STUDY WHICH RECEIVES CORE CLINICAL DATA ELEMENTS FROM SOME CARE CENTERS. CAREPPMD'S CARE PROGRAM STRIVES TO ENSURE THAT PEOPLE LIVING WITH DUCHENNE ARE LIVING LONGER, STRONGER LIVES, BY HELPING PROVIDE ACCESS TO EXPERT HEALTHCARE PROVIDERS, A COMPREHENSIVE TEAM OF SUB-SPECIALISTS, AND APPROVED TREATMENTS. PPMD IDENTIFIES GAPS IN CARE FOR PEOPLE WITH DUCHENNE THROUGHOUT THE LIFESPAN AND COLLABORATES WITH HEALTH CARE PROFESSIONALS ACROSS THE GLOBE TO IMPROVE HEALTH OUTCOMES FOR ALL DUCHENNE PATIENTS. PPMD'S CERTIFIED DUCHENNE CARE CENTER PROGRAM (CDCC) HELPS TO ENSURE THAT CENTERS COMPLY WITH THE STANDARDS OF CARE AND SERVICES THAT HAVE BEEN ESTABLISHED IN THE DUCHENNE CARE GUIDELINES. ALL CERTIFIED DUCHENNE CARE CENTERS HAVE MET THE REQUIREMENTS FOR, AND AGREE TO PROVIDE, STANDARDIZED CARE AND SERVICES.

  Expenses $7,116,727 · Grants $2,560,127 · Revenue $1,262,521

• EDUCATIONPPMD'S EDUCATION INITIATIVES INCREASE AWARENESS AND UNDERSTANDING OF THE DISEASE PROGRESSION AND THE COMPONENTS OF MULTIDISCIPLINARY COMPREHENSIVE CARE NECESSARY TO OPTIMIZE QUALITY AND QUANTITY OF LIFE THROUGHOUT THE LIFESPAN. PPMD PARTNERS WITH EXPERTS IN DUCHENNE TO DEVELOP UP-TO-DATE INFORMATIONAL RESOURCES REGARDING TREATMENT AND CARE THAT WE THEN SHARE BROADLY. PPMD COLLABORATES WITH NATIONAL AND INTERNATIONAL ORGANIZATIONS TO DEVELOP AND DISSEMINATE EDUCATIONAL RESOURCES APPROPRIATE FOR THE GLOBAL DUCHENNE COMMUNITY.ENGAGEMENT PPMD BRINGS PEOPLE TOGETHER FOR SUPPORT AND LEARNING OPPORTUNITIES AT ALL TIMES. PPMD HOSTS THE LARGEST GLOBAL DUCHENNE CONFERENCE EVERY SUMMER, BRINGING TOGETHER PATIENTS, FAMILIES, CLINICIANS, RESEARCHERS, AND INDUSTRY PARTNERS FROM ACROSS THE GLOBE TO SHARE THE LATEST NEWS AND UPDATES. PPMD ALSO CONVENES MEMBERS OF THE DUCHENNE COMMUNITY THROUGH DIFFERENT GROUP GATHERINGS - VIRTUALLY AND IN PERSON AT VARIOUS POINTS THROUGHOUT THE YEAR. CURRENT GROUPS INCLUDE DUCHENNE CARRIERS, DUCHENNE SIBLINGS, DUCHENNE DADS, DUCHENNE GRANDPARENTS AND NEWLY DIAGNOSED GROUPS. PPMD ALSO OFFERS 1:1 CONNECTION BASED ON SPECIFIC NEEDS THROUGH PPMD TOGETHER - VIRTUAL RESOURCE MEETINGS.

  Expenses $1,933,388

• ADVOCACYPPMD'S ADVOCACY EFFORTS ARE FOCUSED ON ADVANCING CARE AND TREATMENTS FOR DUCHENNE BY LEVERAGING FEDERAL RESOURCES, BUILDING PARTNERSHIPS, AND ADVANCING REGULATORY PROCEDURES AND INFRASTRUCTURE. WE EMPOWER ADVOCATES WITH THE TOOLS AND INFORMATION TO ENGAGE WITH CONGRESSIONAL REPRESENTATIVES TO FOSTER DUCHENNE CHAMPIONS WITHIN CONGRESS AND TO SUPPORT LEGISLATION AND REGULATORY POLICIES, ENSURING THAT DUCHENNE PRIORITIES ARE REFLECTED ACROSS ALL GOVERNMENT AGENCIES.PPMD ALSO LEADS EFFORTS FOR FEDERAL AND LOCAL LEGISLATION TO SUPPORT NEWBORN SCREENING FOR DUCHENNE.

  Expenses $1,137,597

As filed on the 2023 return. The filer continues this part on Schedule O, which is not shown here.

Listed in Part VII

Compensation is as filed in Part VII: reportable pay from this organization and from related organizations, other compensation, and hours per week. The total, marked calculated, is our sum of the three dollar columns, with a blank column counted as $0; the form itself has no total. On a Form 990-EZ the columns are Part IV's instead, and on a Form 990-PF Part VIII's — compensation, contributions to benefit plans, and expense account — and the total sums those. A Schedule J breakdown is as filed: base pay, bonus, other, deferred compensation and nontaxable benefits from this organization, with any from related organizations beside each, and the filer's own total. It is shown with the Part VII listing of the same name on the same return. The form gives no key between the two, so that pairing is ours, and is marked matched by us.

Schedule J

Part I's answers as filed. A line not listed was left unanswered; on lines 1a and 3 that means the box was not checked.

Grants on this return

• CINCINNATI CHILDREN'S HOSPITAL

  $588,811

  RESEARCH - ACTION NETWORK GRANT, RESEARCH- SIBLING CASE REPORTS, CARE PROGRAM- AWARD FOR MAINTAINING PPMD CERTIFIED DUCHENNE CARE CENTER STANDARDS.

• REGENTS OF THE UNIVERSITY OF CALIFORNIA -LA

  $310,000

  RESEARCH- DMD MOLECULAR AND CELLULAR DYNAMICS AT SINGLE NUCLEI RESOLUTION & WELLSTONE SUPPLEMENT

• UNIVERSITY OF FLORIDA FOUNDATION

  $305,346

  RESEARCH- PRECLINICAL THERAPEUTIC ASSESSMENT LAB AND UF BIOREPOSITORY SUPPORT- LILLY, NCH, CATABASIS SAMPLES STORAGE

• UNIVERSITY OF FLORIDA

  $275,765

  RESEARCH- WELLSTONE SUPPLEMENT AND CLINICAL STUDY OF ANTI-AAV ANTIBODY DEPLETION

• TEAM JOSEPH

  $140,000

  CARE PROGRAM- EXCEPTIONAL NEEDS GRANT

• NORTHWESTERN UNIVERSITY

  $110,250

  RESEARCH- WELLSTONE SUPPLEMENT

• NEW YORK UNIVERSITY

  $100,000

  RESEARCH- PEDIATRIC GENE THERAPY MEDICAL ETHICS

• CRITICAL PATH

  $99,601

  RESEARCH- SUPPORT OF DUCHENNE REGULATORY SCIENCE CONSORTIUM

• THE NEMOURS FOUNDATION

  $80,003

  CARE PROGRAM- AWARD FOR MAINTAINING PPMD CERTIFIED DUCHENNE CARE CENTER STANDARDS

• RESEARCH FOUNDATION FOR SUNY (BINGHAMTON)

  $75,000

  RESEARCH- TARGETING THE INNATE IMMUNE SYSTEM TO BLOCK ACUTE INFLAMMATORY AND CHRONIC IMMUNE RESPONSES TO TRANSGENE AND AAV VECTOR IN DMD-2021

The 10 largest by cash amount. The API serves every itemized grant.

Provenance

Return type

form_990

Tax year

2023

Filed

17 July 2024

IRS schema version

2023v5.1

Source object

202412069349300631

Check it against the return as filed: the GivingTuesday Data Lake hosts a public copy of the original XML.

The source document this was parsed from is served over the API, which is where bulk and document access live.