Novel immunotherapeutic interventions have shown promise in harnessing the immune system’s capabilities to target malignant cells.
Rare disease advocacy groups are lobbying Congress to amend regulations to let people with disabilities remain in their wheelchairs during flights.
Three years after COVID-19 was declared a pandemic, rare disease patients still fear the contagion—and must deal with the fallout.
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.