Venture Philanthropy Model

Engages in activities that promote biomedical research, supporting advancements in the field through non-direct research initiatives.

Funds lobbying efforts through O'Neill and Associates to advance policy and funding support for Duchenne Muscular Dystrophy research.

Funds medical and scientific research to improve treatments and find a cure for Duchenne Muscular Dystrophy, including specific projects such as the Phase I/IIa Follistatin Gene Therapy Trial and the "Of Mice and Measures" study. The organization has directed over $2.6 million toward research efforts.

Provides grants to support the operations of specialized clinics, such as the UMass Memorial Duchenne Clinic, to ensure access to care for individuals with Duchenne Muscular Dystrophy.

Organizes annual events including the Step for a Cure 5K Run & Walk, Spring Dinner Dance Gala, Disco Deck Party, and teams for the NYC Marathon and NYC Half Marathon to raise awareness and funds. The 2024 Step for a Cure event engaged over 1,500 participants.

Provides benevolent support including funding for accessible bathrooms, ceiling lifts, pool lifts, accessible vans, and other medical equipment to improve quality of life for individuals with Duchenne Muscular Dystrophy.

Offers information about Duchenne Muscular Dystrophy, including its genetic basis, progression, and impact on mobility and respiration, to increase public understanding.

Conducts and supports scientific research on melanoma, vitiligo, psoriasis, antimicrobial peptides, skin stem cell function, wound healing, and skin cancer, contributing to improved understanding of diagnosis, treatment, prognosis, and potential cures.

Awards grants to support research on melanoma, vitiligo, psoriasis, and other skin diseases, with over $50 million committed and more than $1 million in annual grant funding provided to early-career and established scientists. Has supported over 300 young scientists and dermatology investigators.

Runs a long-standing Research Scholars program that supports early-career dermatology researchers through training and professional development opportunities, operating for over 16 years.

Offers free public education on skin health through a hotline, website, publications, and educational programs such as "The Wonders of Skin" for K-12 and "The Skin You're In" distributed to 15,000 middle schools. Has reached over 12 million young people, teachers, and parents with sun safety and skin health education.

Presents the Founders Award and Research Achievement Awards at professional dermatology events such as the Society for Investigative Dermatology to recognize excellence in research.

Develops expertise and collaborative networks to advance solutions for underserved communities in pediatric healthcare, including launching a fellowship program and participating in the ARPA-H Investor Catalyst Hub Spoke Network to strengthen innovation ecosystems.

Supports and drives research initiatives that develop new approaches for treating rare congenital disorders and pediatric diseases, including catalyzing research on SARM1 inhibitors for rare eye diseases and advancing therapeutic programs through donated funding.

Advances pediatric medical devices and drug therapies through partnerships with industry, experts, and philanthropic organizations, focusing on early-stage innovation and unmet needs in pediatric and rare diseases. Activities include de-risking technologies for clinical application and expanding a portfolio of treatments for pediatric populations.

Provides grants and funding to accelerate research, early clinical development, and clinical collaborations for pediatric medical devices and therapeutics, including support for clinical trials and development-to-market pathways. Includes funding for specific research on rare diseases such as ADOA and retinoblastoma.

Organizes and hosts events such as the Impact Series to convene stakeholders and accelerate pediatric innovation, exemplified by the February 25, 2026 event in New York City titled "Children Can’t Wait."

Runs a charitable organization that combines for-profit leadership and infrastructure with non-profit mission goals to eliminate healthcare barriers, particularly in pediatric and rare disease innovation.