Two Families Revolutionize Rare Disease Advocacy in Massachusetts

News Summary

In Cambridge, Massachusetts, two families, the Dions and Baileys, are transforming rare disease advocacy through their unwavering persistence and collaboration. Focusing on Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), they emphasize collective patient advocacy, showcased at the 2025 Patient Advocacy Summit. Their journey highlights the power of community partnerships in driving research and improving lives, proving that love can indeed change science and inspire real results in rare disease care.

Families Spark a Revolution in Rare Disease Advocacy

In the vibrant biotechnology hub of Cambridge, Massachusetts, an extraordinary movement is taking shape, led by two inspiring families. Their unwavering persistence and incredible collaboration are fundamentally transforming how patient advocacy works for rare diseases. The Dion and Bailey families, whose children face the challenges of Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), recently took center stage at the Massachusetts Biotechnology Council’s (MassBio) 2025 Patient Advocacy Summit. Their remarkable journey is creating a new blueprint, showing just how powerful communities and industry partners can be when they work together to speed up breakthroughs and deliver real results for patients.

A Unified Stand Against a Rare Condition

At the heart of this advocacy revolution are the Dion and Bailey families. Courtney and Joe Dion’s challenging path began in the summer of 2022 when their eldest son, Peter, started having trouble with everyday movements. Tests soon confirmed the diagnosis of LGMD2C, a serious type of muscular dystrophy that often appears in childhood. Not long after, their youngest daughter, Maggie, received the same diagnosis. Chrissy and Brian Bailey’s daughters, six-year-old Berkley and eight-year-old Kennedy, went through nearly a year of incorrect diagnoses and dismissals before blood tests revealed very high Creatine Kinase (CK) levels. These parallel stories of heartache and determination have forged an incredibly strong partnership.

Together, the Dions and Baileys lead the Dion Foundation, an advocacy organization that has pioneered an incredibly innovative model for working together. This unique structure allows families to come together under one shared umbrella, featuring both a general fund and individual family funds. This approach smartly recognizes that each journey with LGMD is unique, while still fostering a powerful collective purpose. This spirit of collaboration, fueled by relentless hope, is crafting a new way forward for rare disease advocacy. It’s a model that effectively brings together families, researchers, and the biotechnology industry around a common mission: ensuring that no child is ever defined by their diagnosis.

Celebrating Progress and Inspiring Change

The 2025 MassBio Patient Advocacy Summit provided the perfect stage to share these compelling stories. Nine-year-old Maggie Dion delivered an incredibly powerful address, articulating her vision for a future where no child suffers from a curable disease. She emphasized that “Love can change science, and families can change the world,” leaving everyone in attendance both thoughtful and energized. Her presence clearly highlighted the very human element at the core of rare disease advocacy.

The summit’s theme, “Purpose, In Spite of Pressure,” truly highlighted the deep commitment to making breakthroughs happen, even amidst the immense challenges often found in biotech and healthcare. For professionals in the industry, this means always keeping patients at the center as market forces evolve. For advocates and researchers, it transforms personal challenges into broad systemic change. For patients and their families, it means finding strength in shared stories and collective action. The direct impact of patient advocacy was crystal clear in Maggie’s recent experience. After six months on a clinical trial, her legs no longer hurt. And Peter, who once tired easily, can now play outside for much longer periods. These personal successes are powerful examples of how patient leadership directly fuels progress in such a meaningful way.

MassBio’s Push for Systemic Solutions

The Massachusetts Biotechnology Council (MassBio), based in Cambridge, Massachusetts, actively supports patient advocacy. They do this by bringing patient stories to the forefront through important events like the Patient Advocacy Summit and Rare Disease Day. Beyond these inspiring platforms, MassBio’s policy initiatives also play a truly critical role. They focus on securing national priorities such as preserving orphan drug incentives and restoring full Research and Development (R&D) expensing. These crucial measures are specifically designed to encourage vital follow-on investment into rare disease research and allow newer companies to immediately deduct their R&D costs, thereby fostering innovation and improving accessibility for patients who desperately need it.

Understanding the Wider Rare Disease Community

The immense challenges faced by families like the Dions and Baileys are sadly widespread within the larger rare disease community. On average, individuals living with rare diseases often experience a waiting period of five years between their first symptoms and finally receiving an accurate diagnosis. More than half of these patients wait at least six months after their initial medical consultation before they get the answers they need. Globally, approximately one in every ten people lives with a rare disease diagnosis, which truly underscores the broad impact of these conditions. Rare diseases affect nearly 30 million Americans and countless others worldwide, emphasizing the critical and ongoing need for continued advocacy and research.

Diverse Efforts Across the Nation

Across the United States, various organizations are driving significant advocacy efforts. In Michigan, the MI-RARE (Michigan Rare Coalition) is dedicated to improving the quality of life for all Michigan residents affected by rare conditions. They hosted the Michigan Rare Disease State Advocacy Day at the Capitol on September 30, 2025, offering a crucial opportunity for patients, families, and caregivers to engage directly with state legislators and advocate for important rare disease issues. This initiative, a partnership with the EveryLife Foundation for Rare Diseases, focuses intensely on awareness, advocacy, and education at the state level.

The Bonnell Foundation is another prominent advocate, specifically for patients with Cystic Fibrosis (CF). Founded by Laura Bonnell, whose two adult daughters live with CF, the foundation works tirelessly to raise awareness about the disease and address serious concerns regarding prescription drug affordability boards, which could potentially harm research and development efforts. This foundation also provides essential support for the day-to-day expenses associated with living with CF.

On a national level, the Patient Advocate Foundation (PAF) provides essential services to Americans nationwide who are living with chronic, life-threatening, and debilitating illnesses. Established in 1996, PAF offers professional case management services, including free and confidential navigational assistance with the often-complex financial and practical challenges that can impact healthcare access. PAF received the 2024 RDDC Patient Advocacy RISE Award, recognizing its steadfast commitment and outstanding efforts in addressing the unique challenges faced by those affected by rare diseases.

For individuals with extremely rare conditions, the n-Lorem Foundation, a non-profit organization based in California, focuses on developing and providing personalized experimental Antisense Oligonucleotide (ASO) medicines for nano-rare patients. The term nano-rare refers to genetic mutations found in only 1 to 30 patients globally. The n-Lorem Foundation commits to providing these individualized treatments for free, for life. As of late 2024, n-Lorem had treated 15 patients and projected to treat over 25 by the end of that year, demonstrating significant progress in this specialized field. The foundation also held its second annual Nano-rare Patient Colloquium in Cambridge, Massachusetts, on October 30-31, 2024, bringing together patients, physicians, and partners.

In the biopharmaceutical industry, companies like Gilead Sciences contribute significantly to addressing unmet medical needs. Headquartered in Foster City, California, Gilead focuses on researching and developing antiviral drugs for conditions such as HIV/AIDS, hepatitis B, hepatitis C, influenza, and COVID-19. Their research scholars program supports new investigations into rare liver disease, reflecting a broader commitment to scientific innovation in areas of high unmet need.

The Future is Bright for Patient-Driven Innovation

The inspiring efforts of families like the Dions and Baileys, alongside dedicated organizations and industry partners, truly underscore a powerful truth: when patients and their advocates take the lead, incredible progress is possible. Their collaborative spirit is not just changing individual lives; it’s reshaping the entire landscape of rare disease research and care, promising a brighter future for countless individuals affected by these challenging conditions.

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