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PEDIATRIC NEUROTRANSMITTER DISEASE ASSOCIATION INC
FARMINGDALE, NY · EIN 113450441 · Form 990 · FY2021 · Micro (<$100K) · pndassoc.org
revenue
$2K
expenses
$216K
net assets
$0
employees
0
program ratio
99%
mission · from form 990

THE ORGANIZATION USES EXISTING FUNDS AND RAISES ADDITIONAL FUNDS TO SUPPORT RESEARCH IN PEDIATRIC NEUROTRANSMITTER DISEASES AND PROVIDES SUPPORT TO FAMILIES AFFECTED BY PNDS

profile · synthesized from sources

Pediatric Neurotransmitter Disease Association supports research into rare pediatric neurotransmitter diseases and provides support to affected families. The organization focuses on improving diagnosis, advancing treatments, and advocating for children with conditions such as tyrosine hydroxylase deficiency and AADC deficiency. It operates as a patient advocacy and support network based in New York.

irs program accomplishments · form 990 part iii · fy2021

What they reported doing

  1. #1 primary $221
    MAINTAIN WEBSITE TO ALLOW AFFECTED FAMILIES TO MAKE CONTACTS FOR ADVISE AND SUPPORT
  2. #2 $165K
    THE ORGANIZATION DONATED ALL OF IT'S ACCUMULATED CASH TO OTHER NOT FOR PROFIT ORGANIZATIONS PERSUANT TO A PLAN OF DISSOLUTION IN ACCORDANCE WITH THE ENTITY BYLAWS. SEE SCHEDULE N
named programs · 4 · from sources

What they call their work

AADC Gene Therapy Clinical Trial
Clinical trial at UCSF evaluating targeted gene transfer for children with AADC deficiency, supported and advocated by PND Association
International Registry on Neurotransmitter Related Disorders (iNTD)
Observational study to describe the natural history and long-term manifestations of neurotransmitter disorders and develop evidence-based guidelines for diagnosis and treatment
J.J. Speller PND Testing Fund
Provides support for diagnostic testing for pediatric neurotransmitter diseases in honor of J.J. Speller
PND Family and Physician Registries
Maintains registries to connect affected families and physicians specializing in pediatric neurotransmitter diseases
activities · 5 groups

What they do

  • Rare Genetic Disease Research and Advocacy 3 activities
    • Disseminating educational and scientific resources
      Produces and distributes printable educational guides, maintains updated scientific references, and provides disease-specific informational resources on genetic disorders affecting neurotransmitter synthesis and metabolism in children.
    • Operating family and physician registries
      Maintains a family registry for families affected by pediatric neurotransmitter diseases (PND) and a physician registry for healthcare providers involved in PND care, supporting research and clinical coordination.
    • Providing clinical guidance to healthcare professionals
      Offers guidance to physicians and consultants on laboratory testing for pediatric neurotransmitter diseases, including Tyrosine Hydroxylase deficiency, to improve diagnostic accuracy and clinical management.
  • Genetic and Neurological Disease Research Funding 3 activities
    • Funding research on pediatric neurotransmitter diseases
      Funds research initiatives including gene therapy, mouse model development, and clinical trials for pediatric neurotransmitter diseases, with specific grants awarded to researchers at institutions such as Emory University, Columbia University, UCSF, and Mount Sinai. Total grants awarded include up to $107,500 in direct funding for research projects.
    • Supporting clinical development of gene therapy for AADC deficiency
      Funded and committed financial support for the AADC gene therapy clinical trial between 2010 and 2013, including $70,000 in funding and an additional $10,000 commitment, to advance regulatory and clinical development efforts.
    • Supporting development of animal models for neurotransmitter diseases
      Funded the creation of the first and only mouse model of Tyrosine Hydroxylase (TH) deficiency at Emory University in 2006, including continued support for research using this model to study dopamine receptor subtypes and movement disorders.
  • Patient and Researcher Conferences 2 activities
    • Advocating for pediatric neurotransmitter disease research and policy
      Attends and participates in the Parkinson's Action Network Research & Public Policy Forum annually since 2007 to advocate for research and public policy advancements relevant to dopamine-related pediatric neurotransmitter diseases.
    • Organizing and supporting scientific symposia on pediatric neurotransmitter diseases
      Organized the First Annual Symposium on Pediatric Neurotransmitter Diseases in 2003, co-presented a medical symposium on multidisciplinary management, and facilitated publication of proceedings in peer-reviewed journals such as the Journal of Inherited Metabolic Disease and Annals of Neurology Supplement.
  • Organization Website and Online Platform Management 1 activity
    • Facilitating peer and professional support networks
      Operates restricted online email support groups for registered family members via Yahoo Groups and maintains a website to enable families to connect for advice and support.
  • Diagnostic Testing and Imaging Services 1 activity
    • Providing diagnostic support and testing resources
      Provides information on diagnostic testing methods, offers a two-stage testing procedure for AADC deficiency, and supports blood sample collection and shipping guidance. Also connects physicians with specialized laboratories and experts to facilitate accurate diagnosis.
financials · form 990 · fy2021
revenue
Total revenue$2K
Contributions & grants$2K100%
Program service revenue$00%
Investment income$00%
Other revenue$0
expenses
Total expenses$216K
Program expenses99%
Admin / overhead1%
Fundraising0%
Salaries & benefits$0
Grants paid out$0
Largest expense lineProfessional Fees
balance sheet
Total assets$0
Cash—
Investments—
Liabilities—
Net assets$0
2 years on record · 2020–2021
leadership · form 990 part vii · fy2021

Who runs it

board members · 4
  • DARLENE DREW — DIRECTOR
  • JOHN SPELLER — TREASURER/DI
  • KYLER DREW — DIRECTOR
  • NANCY SPELLER — PRESIDENT/DI
relationships · 26

Who they work with

  • Annals of Neurology Network — Published proceedings from the First Annual Symposium on Pediatric Neurotransmitter Diseases in its supplement series.
  • Baylor University Medical Center Institute of Metabolic Disease Partner — Collaborating institution providing expertise in metabolic disease and neuropharmacology for diagnostic support.
  • Baylor University Medical Center Institute of Metabolic Disease Partner — Partners with the Pediatric Neurotransmitter Disease Association to offer specialized diagnostic services and research support.
  • Columbia University Medical Center Partner — Hosted grant recipient Dr. Umrao Monani for research on AADC deficiency.
  • Emory University Partner — Collaborates with Dr. Ellen Hess' laboratory to study TH deficiency and develop mouse models.
  • Emory University Partner — Funded research at Emory University to study TH deficiency and develop a mouse model.
  • Emory University School of Medicine Partner — Hosted grant recipient Dr. Ellen J. Hess for analysis of dopamine signaling defects.
  • Genetic Alliance Network — Member of the Genetic Alliance network of disease organizations.
  • Genetic Alliance Network — Member of the Genetic Alliance network.
  • Icahn School of Medicine at Mount Sinai Partner — Hosted grant recipient Dr. Toni Pearson for clinical trial development and natural history characterization.
  • Johns Hopkins School of Medicine Partner — Co-presented a medical symposium on pediatric neurotransmitter disease management
  • Johns Hopkins School of Medicine Partner — Co-sponsored a medical symposium with Johns Hopkins School of Medicine.
  • Medical Neurogenetics, LLC Partner — Collaborates with the Pediatric Neurotransmitter Disease Association to provide diagnostic laboratory support and expertise.
  • Michael J Fox Foundation Partner — Collaborated with the Michael J Fox Foundation on research and advocacy initiatives.
  • National Institute of Neurological Disease and Stroke Partner — Co-presented a medical symposium on pediatric neurotransmitter disease management
  • National Institute of Neurological Disease and Stroke Partner — Co-sponsored a medical symposium with the National Institute of Neurological Disease and Stroke.
  • Office of Rare Diseases Government — Collaborated with the Office of Rare Diseases on a medical symposium.
  • Office of Rare Diseases Partner — Co-presented a medical symposium on pediatric neurotransmitter disease management
  • Other nonprofit organizations Partner — Distributed accumulated cash to other nonprofit organizations as part of a dissolution plan.
  • PND Facts Network — Source of disease-specific information linked by the organization.
  • Parkinson's Action Network Partner — Collaborates with PAN by participating in joint advocacy and policy forums.
  • Parkinson's Action Network Partner — Partnered with Parkinson's Action Network for advocacy and public policy efforts.
  • Teodoro Bottiglieri Partner — Scientific collaborator and contact for laboratory testing guidance on pediatric neurotransmitter diseases.
  • University of California, San Francisco Partner — Hosted grant recipient Dr. Krystof Bankiewicz for gene therapy regulatory preparation.
  • Yahoo Groups Partner — Hosts the PND Association's restricted online email support groups.
  • domestic and international advisory board Partner — Provides guidance through a domestic and international advisory board.
strategies · 5

How they approach the work

Named approaches extracted from this org’s sources. Where others share an approach, follow it to see the full set of orgs running it.

  • Expert-Led Strategic Planning for Research Advancement
    methodology: expert-led strategy planning
    By using expert-led scientific discussions to guide strategy, the organization ensures that research and organizational goals are aligned with cutting-edge science, because involving leading researchers enhances the validity, feasibility, and impact of funded initiatives.
  • Multidisciplinary Collaboration to Develop Clinical Guidelines
    methodology: multidisciplinary_approach
    By convening scientists and clinicians across disciplines, the organization develops practical treatment guidelines for pediatric neurotransmitter diseases, because integrating diverse expertise improves clinical decision-making and standardizes care for rare, complex conditions.
  • Patient and Family Advocacy to Center Lived Experience
    methodology: patient_and_family_advocacy
    By advocating for children and families affected by pediatric neurotransmitter diseases through a patient-centered, family-focused approach, the organization ensures that research and policy reflect real-world needs, because centering lived experience improves relevance and responsiveness of medical and support systems.
  • Targeted Research and Advocacy for Rare Neurotransmitter Diseases
    methodology: targeted_disease_focus
    By focusing research and advocacy efforts on dopamine-related pediatric neurotransmitter diseases, the organization advances understanding, improves treatments, and accelerates progress toward cures, because prioritizing specific biological pathways enables more efficient therapeutic development and policy engagement.
  • Umbrella Classification to Unify Research and Treatment
    methodology: umbrella_classification
    By classifying diverse genetic disorders affecting neurotransmitter synthesis, metabolism, and catabolism under the unified term Pediatric Neurotransmitter Disease (PND), the organization improves research coordination and clinical understanding, because a shared diagnostic and conceptual framework enables cross-disease insights and accelerates therapeutic development.