Recent Articles

Understand the challenges rare disease young patients face when transitioning to adult care and why preparation should begin in early adolescence.

Multidisciplinary clinics for rare diseases unite specialists from different fields to collaborate on diagnosis and treatment, helping patients receive faster answers, coordinated care, and access to advanced therapies."

How researchers prove that a gene causes a disease. A clear guide to the scientific methodology, evidence standards, and validation process used in rare disease research.

For millions living with rare diseases, effective treatments remain out of reach. Learn why drug development takes longer for rare conditions and how innovation is starting to close the gap.

Understanding the difference between prevalence and incidence is key to interpreting health data, planning care, and supporting rare disease communities.

Rare disease care is funded by governments, nonprofits, pharma, and foundations. See who supports research, treatment, and patient programs.

Rare disease research funding is limited but creative. Discover government grants, patient-led support, international alliances, networks, and emerging models driving progress for 7,000+ conditions.

Why is awareness not enough in rare diseases? See the gap between visibility and understanding, and its impact on diagnosis, care, and research.

Gene discoveries are often misunderstood. Learn what a new gene finding really means for diagnosis, research, and patient care in rare diseases.

Rare diseases affect millions worldwide. Let's explore common misconceptions about genetics, treatment access, research, and public health.