At their core, patient organizations (also commonly called patient advocacy groups) are
support networks for individuals and families affected by specific diseases and disorders …

Fifteen years ago, DMM was founded on the idea that 'application of fundamental
discoveries from (model) organisms will accelerate our understanding, diagnosis and …

In the face of inadequate treatments, rare disease patients have begun acting like scientists
and studying themselves. Through online networks, patient groups transform disease …

Background: Patient advocacy groups (PAGs) serve a vital role for rare disease patients and
families by providing educational resources, support, and a sense of community. Motivated …

In this article, the authors review the unique role that patient-driven organizations, such as
patient advocacy groups and voluntary health organizations (PAG/VHOs), play in …

Rare disease patient organisations (RDPOs) provide funding, logistic support and ideas for
biomedical research. The literature, however, largely consists of case studies of successful …

People with rare lysosomal storage diseases face challenges in their care that arise from
disease complexity and heterogeneity, compounded by many healthcare professionals …

Despite over 7000 known rare diseases, reliable information regarding their prevalence,
history and treatment options is scarce. To address this gap, patients, advocates, and …

Plain English summary Patients with rare diseases often help to develop new treatments for
their conditions. But once developed, those treatments are sometimes priced too high for …

Extensive public-private partnerships, including the National Institutes of Health (NIH) and
the rare diseases community, which is seeing a renewed industry interest in smaller niche …