A new report from Golin Health, called “The Raising Rare Report,” reveals a communication gap between young people living with rare diseases like Duchenne muscular dystrophy (DMD) and their caregivers when it comes to preparing for the transition to adulthood.
The study focused on patients between the ages of 16 and 23 and their caregivers, aged 25 to 55. One of the key findings was that 77% of patients said they plan to change their treatment plans once they began managing their own care, while only 53% of caregivers expected any changes. Similarly, 79% of caregivers worry that their loved ones aren’t ready to manage their own health, but 79% of independent patients said they had felt prepared to take charge of their care.
To prepare for the transition, patients said they needed guidance from healthcare providers, the chance to talk to other patients of the same age who understand what they’re going through and mental health support. On the other hand, caregivers prioritize practical preparation like everyday disease management, understanding treatment options, knowing the risks of the condition and building life skills for independence.
The report also highlighted the emotional toll caregiving takes. About 73% of caregivers said their emotional health has been affected, 74% feel overwhelmed, 53% said caregiving has strained their personal relationships and 40% said they feel lonely.
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The study surveyed 261 patients and 120 caregivers between April and June 2025 and included insights across 175 rare diseases.
Patients and caregivers also prefer different information sources, with patients tending to rely on advocacy groups and social media and caregivers preferring treatment websites and materials from healthcare professionals.
Despite these differences, both groups said they trust healthcare providers the most when looking for guidance on the disease and treatment plans. “Real-world insights point to an opportunity — and great need — to empower rare disease doctors to engage their patients as partners and ask about their values and long-term goals so they can discover how medicine can complement their lives instead of commanding them,” the report said.
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