Form 990 for tax year 2025

Totals

Total revenue

$2,674,435

Total expenses

$2,032,838

Contributions and grants received

$2,481,858

Grants paid

$459,015

Program service revenue

$14,946

Investment income

$278,337

Other revenue

-$100,706

Total assets, end of year

$6,181,230

Total liabilities, end of year

$350,810

Net assets or fund balances, end of year

$5,830,420

Program services expenses (Part IX)

$1,563,879

Management and general expenses

$215,812

Fundraising expenses

$253,147

Program service expenses (Part III)

$1,563,879

Program services were 76.9% 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 · 2025

• THE NPKUA APPROVED 5 RESEARCH GRANTS FROM THE 2025 RESEARCH GRANT CYCLE FOR A TOTAL OF $425K. THE RESEARCH PROJECTS FOCUS ON PKU ASSESSMENT TOOLS AS WELL AS PKU TREATMENTS AND THERAPIES THAT DRIVE PROGRESS TOWARD A CURE. AS OF 2025, THE NPKUA HAS AWARDED $6,419,699 IN SUPPORT OF 88 RESEARCH PROJECTS.

  Expenses $659,425 · Grants $424,958

• COMMUNITY ENGAGEMENT CONTINUES TO BE A CORE PILLAR OF THE NATIONAL PKU ALLIANCE. THE COMMUNITY ENGAGEMENT DIRECTOR IS RESPONSIBLE FOR PLANNING AND MANAGING PROGRAMS FOR THE PKU COMMUNITY, INCLUDING A MENTORING PROGRAM, COMMUNITY CALLS, SUPPORT KITS, MATERNAL PKU PATIENT SUPPORT, ADVOCACY EFFORTS AND AN AFFILIATE PROGRAM.THE DIRECTOR SCHEDULES, MANAGES AND ATTENDS VIRTUAL CALLS WITH THE COMMUNITY ACROSS DIFFERENT PKU AGE GROUPS AND PKU CAREGIVER TYPES. 48 CALLS WERE HELD IN 2025 WITH 250 ATTENDEES.IN 2025, THE NPKUA MENTOR PROGRAM GREW TO SUPPORT OVER 45 CAREGIVERS AND INDIVIDUALS WITH PKU THROUGH PERSONALIZED ONE-ON-ONE MENTORING, WITH MENTORS CONNECTING VIRTUALLY AND IN PERSON TO SHARE EXPERIENCES AND DEVELOP SKILLS TO BETTER ASSIST THEIR MENTEES.THE NPKUA ADVOCACY COMMITTEE WORKS ALONGSIDE MEMBERS OF THE BROADER RARE DISEASE COMMUNITY TO ADVANCE EFFORTS AROUND NUTRITION, EQUITY AND OTHER PRIORITIES OF THE PKU COMMUNITY. THE COMMITTEE DEVELOPED AN ADVOCACY MEDIA TOOLKIT TO HELP COMMUNITY MEMBERS SHARE THEIR STORIES. STAFF AND ADVOCACY COMMITTEE VOLUNTEERS HAVE ALSO RESPONDED TO THE TERMINATION OF THE ACHDNC (ADVISORY COMMITTEE ON HERITABLE DISORDERS IN NEWBORNS AND CHILDREN) BY CALLING ON CONSTITUENTS, THROUGH DATABASE-WIDE EMAIL AND SOCIAL MEDIA OUTREACH, TO SIGN-ON TO CONTACT THEIR LEGISLATORS AND SHARE THEIR STORY AND SUPPORT OF NBS. STARTED A NEWBORN SCREENING VIDEO SERIES TO BE USED IN SEPTEMBER, RECOGNIZED AS NEWBORN SCREENING AWARENESS MONTH. THE SERIES RAISES AWARENESS ABOUT THE IMPORTANCE OF NEWBORN SCREENING, AND THE CRITICAL ROLE OF NEWBORN SCREENING IN IDENTIFYING SERIOUS CONDITIONS THAT CAN LEAD TO SEVERE DISABILITY OR DEATH IF LEFT UNDIAGNOSED. IN TOTAL, NPKUA SUPPORTED 10 STATE AND FEDERAL LEGISLATIVE ADVOCACY EFFORTS.IN 2025 NPKUA PROVIDED $34,057 TO NPKUA AFFILIATES TO SUPPORT COMMUNITY EVENTS AND CAMPS IN THEIR REGIONS. DURING THE YEAR, NPKUA ALSO MET ONE-ON-ONE WITH INDIVIDUAL AFFILIATE LEADERS TO BETTER UNDERSTAND THEIR GOALS AND NEEDS. IN 2025, 206 NEWBORN AND KINDERGARTEN KITS WERE DELIVERED TO FAMILIES WELCOMING A NEW BABY WITH PKU OR SUPPORTING A NEW KINDERGARTENER WITH PKU.IN 2025, 55 PACKAGES OF DIET-APPROPRIATE FOOD WERE DELIVERED TO MATERNAL PKU PATIENTS TO BEST SUPPORT THEIR LOW-PROTEIN DIET.

  Expenses $497,406 · Grants $34,057 · Revenue $28,375

• Withheld by Nonprofiteer: this text may name a private individual.

  Expenses $138,267

• NPKUA HOSTED THE TRUEPKU INITIATIVE TO SHARE THE PKU EXPERIENCE WITH U.S. FOOD AND DRUG ADMINISTRATION (FDA) STAFF, PEOPLE WORKING IN LIFE SCIENCE COMPANIES, HEALTH CARE PROFESSIONALS AND ACADEMIC RESEARCHERS TO HELP SHAPE THE DEVELOPMENT OF NEW MEDICAL THERAPIES AND APPROACHES TO TREATING AND MONITORING PKU. AS PART OF THE EXTERNALLY-LED PATIENT-FOCUSED DRUG DEVELOPMENT (EL-PFDD) PROGRAM, THIS MEETING TOOK PLACE VIRTUALLY ON MAY 8, 2025 IN PARTNERSHIP WITH OTHER PKU-RELATED ORGANIZATIONS AND SPONSORS. 31 MEMBERS FROM THE PKU COMMUNITY SHARED THEIR TESTIMONY. THE VOICE OF THE PATIENT REPORT SUMMARIZES THE MEETING INSIGHTS, PERSONAL STORIES AND KEY THEMES SHARED THROUGHOUT THE FIVE-HOUR MEETING, INCLUDING POWERFUL QUOTES FROM COMMUNITY MEMBERS AND PERSPECTIVES GATHERED BEFORE, DURING AND AFTER THE EVENT. BOTH THE LIVE MEETING AND REPORT CAN BE FOUND AT WWW.NPKUA.ORG/TRUEPKU/. THE MEETING HAS ATTRACTED 1,300 VIEWS ON YOUTUBE.

  Expenses $117,292 · Revenue $10,578

• IN 2025, THE NPKUA HOSTED A REGIONAL COMMUNITY CONFERENCE ON SEPTEMBER 6 IN SAN DIEGO, CALIFORNIA. ATTENDEES INCLUDED 47 INDIVIDUALS WITH PKU, THEIR FAMILIES/CAREGIVERS, INDUSTRY PARTNERS, AND LEADING EXPERTS IN THE FIELD OF PKU RESEARCH, CLINICAL CARE, NUTRITION, AND ADVOCACY. IN TOTAL, WE WELCOMED 180 ATTENDEES. WE HELD MEANINGFUL CONVERSATIONS THROUGH EDUCATIONAL PRESENTATIONS AND ACTIVITIES DISCUSSING WHAT'S NEW IN PKU, STAYING ACTIVE WITH PKU, CLINICAL TRIALS, FOOD AND FORMULA OFFERINGS, GENE EDITING AND GENE THERAPY, TRAVELING WITH PKU AND HOME PHE MONITOR DEVELOPMENT.

  Expenses $83,918 · Revenue $4,368

• IN THE PKU PATIENT REGISTRY, NPKUA UPGRADED THE PLATFORM, STRENGTHENING THE REGISTRY'S ABILITY TO SUPPORT PKU RESEARCH. REGISTRY PARTICIPANTS GREW TO 1,130, WITH 864 NEW SURVEY SUBMISSIONS IN 2025. THE REGISTRY SUPPORTED RECRUITMENT FOR 4 EXTERNAL RESEARCH STUDIES AND SHARED DATA IN ITS FIRST SCIENTIFIC PUBLICATION.

  Expenses $67,571

As filed on the 2025 return. The filer continues this part on Schedule O, which is not shown here. A text marked withheld matched a screen for wording that may name a private individual, such as an honorific or "in memory of". Most of what it withholds names no one; it is withheld so that what does is never shown.

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

• OREGON HEALTH & SCIENCE

  $169,344

  PKU RESEARCH

• UNIVERSITY OF CALIFORNIA SAN DIEGO

  $80,000

  PKU RESEARCH

• BOSTON CHILDREN'S HOSPITAL

  $71,394

  PKU RESEARCH

• EMORY UNIVERSITY SCHOOL OF MEDICINE

  $50,000

  PKU RESEARCH

• MARYLAND ALLIANCE OF PKU FAMILIES INC

  $10,098

  PKU RESEARCH

• PKU ORGANIZATION OF ILLINOIS

  $8,531

  PKU RESEARCH

• CALIFORNIA COALITION FOR PKU AND ALLIED DISORDERS

  $7,500

  PKU RESEARCH

• GEORGIA PKU CONNECT INC

  $7,500

  PKU RESEARCH

Provenance

Return type

form_990

Tax year

2025

Filed

8 May 2026

IRS schema version

2025v4.1

Source object

202611319349303626

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