News Summary
Two families from Massachusetts, the Dions and Baileys, are transforming rare disease patient advocacy by showcasing collaborative efforts in biotechnology. Their focus on Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C) was highlighted at the MassBio 2025 Patient Advocacy Summit, emphasizing the importance of partnerships between families, researchers, and industry to improve patient outcomes. Their innovative approach through the Dion Foundation fosters unity and hope, aiming to connect and inspire the rare disease community.
Cambridge, Massachusetts – Two families from Massachusetts are profoundly impacting rare disease patient advocacy, showcasing the power of sustained effort and collaboration within the state’s biotechnology sector. The Dion and Bailey families, whose children are living with Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), were a central focus at the Massachusetts Biotechnology Council’s (MassBio) 2025 Patient Advocacy Summit, which took place on December 11, 2025, in Cambridge, Massachusetts. Their work illustrates a successful new approach for how rare disease communities and industry partners can unite to speed up innovation and deliver meaningful outcomes for patients.
Families Forge a Collaborative Path
The driving force behind this advocacy shift is the shared experience of the Dion and Bailey families. Courtney and Joe Dion’s journey began in the summer of 2022 when their oldest son, Peter, showed signs of difficulty with common movements, leading to a diagnosis of LGMD2C, a severe childhood-onset form of muscular dystrophy. Soon after, their youngest daughter, Maggie, also received the diagnosis. Similarly, Chrissy and Brian Bailey’s daughters, six-year-old Berkley and eight-year-old Kennedy, faced nearly a year of misdiagnoses before bloodwork confirmed significantly elevated Creatine Kinase (CK) levels. These parallel experiences of challenge and resolve have created a strong partnership between the families.
Together, the Dions and Baileys lead the Dion Foundation, an advocacy organization that has introduced an innovative model for joint action. This model allows families to come together under a unified umbrella, featuring both a general fund and individual family funds. This structure recognizes the unique aspects of each LGMD journey while fostering a collective purpose. This cooperative spirit, combined with unwavering hope, is establishing a new framework for rare disease advocacy, effectively connecting families, researchers, and the biotechnology industry to a common goal: ensuring that no child is defined by their diagnosis.
Celebrating Progress at the MassBio Summit
The 2025 MassBio Patient Advocacy Summit served as a key platform to highlight these powerful stories. Nine-year-old Maggie Dion delivered a moving speech, sharing her vision for a future where no child suffers from a curable disease and emphasizing that “Love can change science, and families can change the world”. Her presence underscored the essential human element in rare disease advocacy, deeply affecting and inspiring those in attendance.
The summit, themed “Purpose, In Spite of Pressure,” emphasized the dedication required to achieve breakthroughs even amid the significant challenges in biotech and healthcare. For professionals in the industry, this means keeping patients at the forefront as market dynamics evolve. For advocates and researchers, it involves transforming personal challenges into broader systemic change. For patients and their families, it means drawing strength from shared experiences and collective action. The immediate impact of patient advocacy was clear in Maggie’s recent improvements; after six months on a clinical trial, her legs no longer cause her pain. Peter, who previously experienced easy fatigue, can now play outside for longer periods. These personal successes demonstrate how patient leadership directly contributes to progress.
MassBio’s Role in Systemic Advocacy
The Massachusetts Biotechnology Council, located in Cambridge, Massachusetts, actively champions patient advocacy by promoting patient stories through events such as the Patient Advocacy Summit and Rare Disease Day. MassBio’s policy initiatives are also vital, focusing on securing national priorities like preserving orphan drug incentives and reinstating full Research and Development (R&D) expensing. These measures aim to encourage crucial subsequent investment into rare disease research and allow emerging companies to immediately deduct R&D costs, thereby promoting innovation and accessibility for patients.
Broader Efforts Across the Rare Disease Community
Understanding the Challenges of Rare Diseases
The difficulties encountered by families such as the Dions and Baileys are common throughout the rare disease community. On average, individuals living with rare diseases endure a waiting period of five years between their first symptoms and receiving an accurate diagnosis. More than half of these patients wait at least six months after their initial medical consultation before getting answers. Globally, roughly one in every ten people lives with a rare disease diagnosis, highlighting the widespread impact of these conditions. Rare diseases affect nearly 30 million Americans and countless others worldwide, underscoring the critical need for ongoing advocacy and research.
State-Level Initiatives for Support and Advocacy
Across the United States, various organizations are driving significant advocacy efforts. In Michigan, the MI-RARE (Michigan Rare Coalition) is committed 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, providing a crucial opportunity for patients, families, and caregivers to engage with state legislators and advocate for important rare disease issues. This initiative, a partnership with the EveryLife Foundation for Rare Diseases, concentrates 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 to raise awareness about the disease and address concerns regarding prescription drug affordability boards, which could potentially hinder research and development. This foundation offers support for the day-to-day expenses associated with living with CF. This foundation’s work has Nationwide impact, with specific state-level advocacy efforts.
National and Global Advocacy Developments
On a national level, the Patient Advocate Foundation (PAF) delivers essential services to Americans with chronic, life-threatening, and debilitating illnesses nationwide. Established in 1996, PAF provides professional case management services, including free and confidential navigational assistance with financial and practical challenges that impact healthcare access. PAF received the 2024 RDDC Patient Advocacy RISE Award, acknowledging its consistent dedication 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. Nano-rare refers to genetic mutations found in only 1 to 30 patients globally. n-Lorem 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. The n-Lorem Foundation’s work has Nationwide / Global reach.
In the biopharmaceutical industry, companies like Gilead Sciences contribute 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. Gilead Sciences has a Worldwide scope.
Frequently Asked Questions (FAQ)
- What rare disease do the Dion and Bailey families advocate for?
- The Dion and Bailey families advocate for patients affected by Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), a severe childhood-onset subtype of muscular dystrophy.
- What is the significance of the 2025 MassBio Patient Advocacy Summit?
- The 2025 MassBio Patient Advocacy Summit, held on December 11, 2025, in Cambridge, Massachusetts, highlighted the stories of families like the Dions and Baileys, showcasing how persistence and collaboration are redefining rare disease advocacy and driving innovation.
- What is the average waiting period for a rare disease diagnosis?
- On average, people living with rare diseases wait five years between their first symptoms and receiving an accurate diagnosis.
- What is the mission of the n-Lorem Foundation?
- The n-Lorem Foundation, a non-profit based in California, discovers, develops, and provides personalized experimental Antisense Oligonucleotide (ASO) medicines for nano-rare patients for free, for life. Nano-rare refers to genetic mutations found in only 1 to 30 patients worldwide.
- What services does the Patient Advocate Foundation (PAF) provide?
- The Patient Advocate Foundation (PAF) is a national non-profit that provides professional case management services and financial aid to Americans with chronic, life-threatening, and debilitating illnesses nationwide, offering free and confidential navigational assistance with financial and practical healthcare access challenges.
- What is the Michigan Rare Disease State Advocacy Day?
- The Michigan Rare Disease State Advocacy Day, hosted by the MI-RARE (Michigan Rare Coalition) on September 30, 2025, is an opportunity for patients, families, and caregivers to advocate for rare disease issues with state legislators in Michigan. This initiative is state-level.
Key Features of Rare Disease Patient Advocacy
| Feature/Organization | Primary Focus | Geographic Scope | Key Contributions |
|---|---|---|---|
| Dion & Bailey Families / Dion Foundation | Advocacy for Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C) | Massachusetts-based initiative, with broader impact model | Redefining patient advocacy, innovative collaborative funding model, direct patient impact (improved health outcomes) |
| Massachusetts Biotechnology Council (MassBio) | Elevating patient stories, advocating for policy changes in biotech | Massachusetts (State-level) | Hosts Patient Advocacy Summit, advocates for orphan drug incentives and R&D expensing |
| MI-RARE (Michigan Rare Coalition) | Improving quality of life for Michigan residents with rare conditions | Michigan (State-level) | Hosts Michigan Rare Disease State Advocacy Day, focuses on awareness, advocacy, and education |
| The Bonnell Foundation | Advocacy for Cystic Fibrosis (CF) patients | Nationwide, with specific state-level advocacy efforts | Raises awareness, addresses prescription drug affordability concerns, provides financial support |
| n-Lorem Foundation | Developing personalized ASO medicines for nano-rare diseases | Nationwide / Global (based in California) | Provides free, individualized experimental ASO treatments for life to patients with genetic mutations found in 1-30 people worldwide |
| Patient Advocate Foundation (PAF) | Case management and financial aid for chronic/debilitating illnesses | Nationwide | Offers free, confidential navigational assistance with healthcare access barriers; received Patient Advocacy RISE Award |
| Gilead Sciences | Biopharmaceutical research and development | Worldwide (Foster City, California headquarters) | Focuses on antiviral drugs, including research into rare liver diseases, addresses unmet medical needs |
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