News Summary
Two Massachusetts families, the Dions and Baileys, are leading a transformative movement in rare disease advocacy for Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C). Their collaborative efforts were highlighted during the MassBio Patient Advocacy Summit, showcasing how teamwork can accelerate innovation in healthcare. This inspiring narrative emphasizes the importance of family-driven solutions and collective action, with a focus on building a supportive community among patients, researchers, and the biotechnology industry.
Massachusetts Families Lead a Rare Disease Revolution: A Story of Hope and Collaboration
In a powerful display of determination and unity, two Massachusetts families are rewriting the playbook for rare disease advocacy. The Dion and Bailey families, united by their children’s diagnoses of Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), are at the forefront of a movement that highlights the incredible impact of persistence and teamwork within the vibrant biotechnology community. Their inspiring journey took center stage at the Massachusetts Biotechnology Council’s (MassBio) 2025 Patient Advocacy Summit, held recently on December 11 in Cambridge, Massachusetts.
This heartwarming story offers a fresh perspective on how rare disease communities and industry partners can join forces to speed up innovation and deliver real, life-changing results for patients. It’s a new model that’s showing the world what’s possible when families refuse to give up.
Building a United Front Against a Tough Foe
The heart of this advocacy breakthrough lies with the courageous Dion and Bailey families. Courtney and Joe Dion’s challenging journey began in the summer of 2022 when their oldest son, Peter, started having trouble with everyday movements. Tests soon confirmed the difficult diagnosis of LGMD2C, a severe form of muscular dystrophy that typically starts in childhood. Not long after, their youngest daughter, Maggie, also received the same diagnosis. Similarly, Chrissy and Brian Bailey’s daughters, six-year-old Berkley and eight-year-old Kennedy, went through almost a year of confusing misdiagnoses and dismissals before blood tests revealed significantly high Creatine Kinase (CK) levels. These parallel experiences of sadness and unwavering resolve have forged an incredibly strong partnership between the two families.
Together, the Dions and Baileys steer the ship of the Dion Foundation, an advocacy group that has introduced a groundbreaking model for working together. This unique structure allows families to rally under one big umbrella, featuring both a general fund and special individual family funds. This approach acknowledges that every family’s journey with LGMD is unique, while also fostering a powerful sense of shared purpose. This collaborative spirit, fueled by endless hope, is truly creating a new blueprint for rare disease advocacy, one that effectively brings together families, researchers, and the biotechnology industry around a common goal: making sure that no child is ever defined solely by their diagnosis.
Cheering on Progress at the MassBio Summit
The 2025 MassBio Patient Advocacy Summit provided the perfect stage to shine a spotlight on these compelling stories. Nine-year-old Maggie Dion delivered a truly moving speech, sharing her heartfelt vision for a future where no child has to suffer from a curable disease. She powerfully emphasized that “Love can change science, and families can change the world.” Her presence beautifully highlighted the human element at the very core of rare disease advocacy, leaving everyone in the audience both quiet and deeply inspired.
The summit’s theme, “Purpose, In Spite of Pressure,” underscored a strong commitment to pushing for breakthroughs even when faced with the enormous challenges inherent in the biotech and healthcare worlds. For professionals in the industry, this means always keeping patients at the center as market forces change. For advocates and researchers, it means turning personal challenges into widespread systemic change. And for patients and their families, it means finding strength in shared stories and collective action. The direct, positive impact of patient advocacy was wonderfully clear in Maggie’s recent experience. After just six months on a clinical trial, her legs no longer hurt, and Peter, who used to get tired easily, can now play outside for much longer. These personal triumphs are shining examples of how patient leadership directly fuels progress and brings about real hope.
MassBio’s Role in Driving Systemic Change
The Massachusetts Biotechnology Council, based in Cambridge, Massachusetts, is a strong supporter of patient advocacy. They actively help by sharing patient stories through important events like the Patient Advocacy Summit and Rare Disease Day. MassBio’s policy initiatives also play a crucial role, focusing on securing national priorities such as protecting incentives for orphan drugs and restoring full Research and Development (R&D) expensing. These measures are designed to encourage vital follow-on investment into rare disease research and allow newer companies to immediately deduct their R&D costs, which in turn fosters innovation and improves accessibility for patients who desperately need new treatments.
Broader Efforts Across the Rare Disease Community
The struggles faced by families like the Dions and Baileys are sadly common within the wider rare disease community. On average, individuals living with rare diseases often experience a five-year waiting period between their first symptoms and finally receiving an accurate diagnosis. More than half of these patients wait at least six months after their initial doctor’s visit before getting answers. Worldwide, about one in every ten people lives with a rare disease diagnosis, which really emphasizes the broad impact of these conditions. Rare diseases affect nearly 30 million Americans and countless others globally, highlighting the critical and ongoing need for continued advocacy and research.
Across the United States, various organizations are making significant strides in advocacy. 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, providing a key opportunity for patients, families, and caregivers to meet with state legislators and champion important rare disease issues. This initiative, a partnership with the EveryLife Foundation for Rare Diseases, focuses on raising awareness, advocating for change, and providing education at the state level.
The Bonnell Foundation is another prominent advocate, specifically for patients living 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 concerns regarding prescription drug affordability boards, which could potentially hurt research and development. This foundation also provides much-needed support for the everyday expenses that come with living with CF.
On a national scale, the Patient Advocate Foundation (PAF) offers essential services to Americans dealing with chronic, life-threatening, and debilitating illnesses nationwide. Established in 1996, PAF provides professional case management services, including free and confidential help navigating the financial and practical challenges that can impact healthcare access. PAF recently received the 2024 RDDC Patient Advocacy RISE Award, recognizing its steadfast commitment and outstanding efforts in tackling the unique challenges faced by those affected by rare diseases.
For individuals with incredibly rare conditions, the n-Lorem Foundation, a non-profit organization based in California, is focused 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. The n-Lorem Foundation makes an incredible commitment to provide these individualized treatments for free, for life. As of late 2024, n-Lorem had already treated 15 patients and expected to treat over 25 by the end of that year, showing significant progress in this highly specialized field. The foundation also held its second annual Nano-rare Patient Colloquium in Cambridge, Massachusetts, on October 30-31, 2024, bringing together patients, doctors, and partners.
In the biopharmaceutical industry, companies like Gilead Sciences contribute significantly to addressing unmet medical needs. Headquartered in 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 where needs are high and current solutions are scarce.
The combined efforts of these families and organizations paint a powerful picture of hope and progress, demonstrating that through collaboration and unwavering dedication, the landscape for rare disease patients is indeed changing for the better.
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