named programs · 3 · from sources
what they call their work
Biomarker Discovery
Program dedicated to identifying biomarkers that can help in the diagnosis and treatment of ASMD.
FAAH Inhibitor Program
Research program focused on developing inhibitors to treat ASMD by addressing endocannabinoid signaling.
Gene Therapy
Invests in gene therapy research aimed at correcting the genetic defects causing ASMD.
activities · 3 clusters
what they do
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ASMD and Niemann-Pick Disease Research Programs 6 activities
- ASMD Accelerate Research StudyConducts the ASMD Accelerate study in partnership with Picnic Health to advance real-world evidence and research on Acid Sphingomyelinase Deficiency (ASMD), including data collection and analysis to support therapeutic development.
- Biomarker and Drug Discovery ResearchConducts biomarker discovery and drug screening research using model organisms and the ASM-KO mouse colony to identify therapeutic targets such as Sphingomyelin 16:0 and understand disease mechanisms in NPA and ASMD.
- FAAH Inhibitor Research ProgramOperates a FAAH inhibitor program investigating FAAH inhibition as a therapeutic approach for neurovisceral acid sphingomyelinase deficiency, including preclinical and translational research.
- Funding and conducting research on Acid Sphingomyelinase Deficiency (ASMD)The foundation advances research programs for Infantile Neurovisceral (NPA) and Chronic Neurovisceral (NPA/B) ASMD to develop treatment options, including operating a FAAH Inhibitor Program, a Gene Therapy program, maintaining an ASM-KO Mouse Colony, and conducting NPA Model Organism Development & Drug Screen research and Biomarker Discovery research. They also develop Real World Evidence (RWE) for ASMD and have launched the ASMD Accelerate study in partnership with Picnic Health.
- Gene Therapy ProgramOperates a gene therapy research program focused on developing treatments for Niemann-Pick disease, including work on adeno-associated viral vector serotype 9-based gene therapy for type A.
- Publishing scientific research on ASMDThe foundation publishes and contributes to scientific publications on ASMD, including case reports on Niemann Pick Disease Type A, research on FAAH inhibition as a therapeutic approach, gene therapy, autopsy pathology, the role of sphingomyelin in neurodegeneration, lipid-induced lysosomal damage, and identifying therapeutic targets for neuronal death in ASMD.
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Cancer-Focused Fundraising & Awareness Events 1 activity
- Living Like A Warrior GalaHosts the annual "Living Like A Warrior" gala event to raise awareness and support for the foundation, with documented events held from 2014 through 2023.
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Uncategorized 17 activities
- ASM-KO Mouse Colony & Model Organism ResearchMaintains an ASM-KO mouse colony and conducts model organism development and drug screening research for NPA to support preclinical therapeutic development.
- ASMD Accelerate Research StudyConducts the ASMD Accelerate study in partnership with Picnic Health to advance real-world evidence and research on Acid Sphingomyelinase Deficiency (ASMD).
- Biomarker Discovery ResearchConducts research to identify and validate biomarkers for ASMD to support diagnosis, monitoring, and treatment evaluation.
- FAAH Inhibition Research ProgramOperates a research program focused on FAAH inhibition as a potential therapeutic approach for neurovisceral acid sphingomyelinase deficiency, including preclinical and publication efforts.
- Facilitating personalized fundraisingThe foundation provides a platform for individuals to create personalized fundraising pages to share their stories and raise funds for the foundation's research programs.
- Gene Therapy Research ProgramOperates a gene therapy research program, including development and testing of adeno-associated viral vector serotype 9-based therapies for Niemann-Pick disease type A.
- Honoring individuals who passed away from ASMDThe foundation recognizes individuals referred to as "Warriors With Wings" who have passed away, listing their names and dates of life.
- Hosting the Living Like A Warrior GalaThe foundation hosts an annual fundraising event called the Living Like A Warrior Gala, which has been held since at least 2014.
- Living Like A Warrior GalaHosts the annual "Living Like A Warrior" gala event to support the foundation’s mission, with documented events from 2014 through 2023.
- Online Inquiry and Contact SupportProvides an online contact form for individuals to submit inquiries or messages to the foundation.
- Personalized Fundraising SupportEnables individuals to create personalized fundraising pages to share their stories and raise funds for the foundation’s research programs.
- Real World Evidence (RWE) Development for ASMDDevelops real-world evidence for ASMD through data collection and research initiatives to inform clinical understanding and therapeutic development.
- Research Advancement for NPA and NPA/BAdvances research programs for Infantile Neurovisceral (NPA) and Chronic Neurovisceral (NPA/B) forms of ASMD to develop treatment options.
- Research Advancement for NPA and NPBAdvances research programs for Infantile and Chronic Neurovisceral Acid Sphingomyelinase Deficiency (Niemann-Pick Types A and B) to develop treatment options, including publication of clinicopathologic findings and therapeutic mechanisms.
- Scientific Publication ContributionsContributes to peer-reviewed scientific publications on Niemann-Pick disease and related lysosomal storage disorders, including pathology, mechanisms of neurodegeneration, and therapeutic targets.
- Warrior With Wings Memorial RecognitionRecognizes individuals referred to as "Warriors With Wings" who have passed away, honoring them by publishing their names and dates of life.
- Warriors With Wings Memorial RecognitionRecognizes individuals with Niemann-Pick disease who have passed away by honoring them as "Warriors With Wings" with their names and dates of life.
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financials · form 990 · fy2022
the money
revenue
Total revenue$551K
Contributions & grants$544K99%
Program service revenue$00%
Investment income$7K1%
Other revenue$52
expenses
Total expenses$179K
Program expenses82%
Admin / overhead10%
Fundraising8%
Salaries & benefits$67K
Grants paid out$63K
Largest expense lineCompensation
balance sheet
Total assets$1.07M
Cash$662K
Investments$405K
Liabilities$1K
Net assets$1.07M
Liquid reserves71.6 mo
3 years on record · 2020–2022 · YoY revenue +0.7%
leadership · form 990 part vii · fy2022
who runs it
paid leadership · 1
| Name | Title | Hours/wk | Compensation |
|---|---|---|---|
| STEVEN LAFFOON | PRESIDENT/TREASURER | 40 | $62K |
board members · 2
- JOE GALLO — DIRECTOR
- MATT COXHEAD — SECRETARY
relationships · 12
who they work with
- Albert Einstein College of Medicine Partner — Scientific and medical advisor affiliated with the organization
- BioVista Funder — Sponsored Wylder Nation Foundation.
- Center of Molecular Biology, Madrid, Spain Partner — Scientific and medical advisor affiliated with the organization
- Children’s Hospital at Montefiore Partner — Scientific and medical advisor affiliated with the organization
- Grace Science Partner — Signed a pact with Grace Science.
- Mount Sinai School of Medicine Partner — Scientific and medical advisor affiliated with the organization
- Perlara Partner — Signed a pact and partnered as a patient advocacy group.
- Pfizer Partner — Scientific and medical advisor affiliated with the organization
- Picnic Health Partner — Partnered to launch the ASMD Accelerate study for advancing ASMD research.
- Picnic Health Partner — Partnered to launch the ASMD Accelerate study.
- UCSF Partner — Received a letter of gratitude from UCSF, implying a collaborative or supportive relationship.
- Wylder Nation Foundation Network — Member of 501(c)3 organizations