Raregivers 100 — Nonprofit Leaders & Global Advocates

The Raregivers 100 honorees in the Nonprofit Leaders & Global Advocates category are driving change at both the systemic and human levels of rare disease. 

These leaders build organizations, influence policy, advance research, and shape global conversations while often drawing on lived experience as patients, parents, or caregivers.

Their work extends beyond individual diagnoses to strengthen the entire rare disease ecosystem and ensure that families are seen, supported, and included across healthcare, education, and society. 

Through persistence and purpose, they transform personal urgency into collective impact, demonstrating how advocacy can move systems, mindsets, and lives.

Nawaal Akram

Nawaal Akram is a disability rights advocate and global voice for inclusion who lives with Duchenne muscular dystrophy. 

Through her work in education and policy, she has challenged stigma around disability and chronic illness, particularly in the Middle East. Her advocacy highlights how rare diseases are shaped not only by biology, but by access, dignity, and societal attitudes.

Brooke Eby

Brooke Eby is a digital creator and ALS advocate who uses humor and honesty to document life with the progressive neurological disease. 

By sharing her diagnosis and daily realities online, she has helped demystify ALS and reduce the discomfort people often feel around serious illness. Her storytelling shows how authenticity can foster empathy and community in the rare disease space.

Pat Furlong

Pat Furlong is the founding president and CEO of Parent Project Muscular Dystrophy and a pioneering leader in rare disease advocacy. 

After her sons were diagnosed with Duchenne muscular dystrophy, she helped build one of the most influential patient-led organizations in the world. Her work demonstrates how caregivers can drive research, policy change, and improved standards of care.

Pamela Gavin

Pamela Gavin is the CEO of the National Organization for Rare Disorders (NORD) and a longtime leader in health advocacy. 

Motivated in part by a family connection to a rare disease, she has focused on reducing diagnostic delays and strengthening the rare disease ecosystem. Her leadership emphasizes collaboration, patient voice, and systemic change across thousands of conditions.

Nikki Lilly

Nikki Lilly is a British advocate and content creator who has shared her life with a rare arteriovenous malformation since childhood. 

By documenting surgeries, recovery, and daily challenges, she has raised awareness about rare conditions and visible differences. Her work underscores the importance of representation and mental health support for young people living with rare diseases.

Tommy Pham

Tommy Pham is a parent advocate who co-founded the Raiden Science Foundation after his son was diagnosed with an ultra-rare genetic disorder caused by UBA5 mutations. 

He has worked directly with researchers to accelerate scientific understanding and the development of potential treatments. His story reflects how families are increasingly becoming catalysts for rare disease research.

Katie Price

Katie Price is a media personality and advocate who has spoken publicly about caring for her son, Harvey, who has Prader–Willi syndrome. 

She has highlighted the long-term health risks, emotional strain, and lack of adult support systems for families affected by rare conditions, using her advocacy to draw attention to the realities of lifelong caregiving.

Paige Rivard

Paige Rivard is a rare disease advocate and caregiver whose child lives with both Prader–Willi syndrome and neurofibromatosis type 1. 

Through leadership roles and public speaking, she’s highlighted the emotional toll of caregiving and the need to support caregivers, reinforcing that caregiver well-being is just as essential to patient outcomes.

Melissa Schlemmer

Melissa Schlemmer is a writer and parent advocate who shares life with her child’s diagnosis of PMM2 congenital disorder of glycosylation.

She writes candidly about medical complexity, daily caregiving, and finding meaning amid uncertainty. Her storytelling highlights the unseen labor and emotional resilience required of families living with rare diseases

Wei Zhang 

Wei Zhang is a researcher working at the forefront of treatments for ultra-rare and nano-rare diseases, including individualized genetic therapies. 

His work focuses on developing targeted approaches for conditions affecting only a handful of patients worldwide, representing the growing movement to ensure even the rarest conditions are not left without hope.