Despite its distinctive clinical presentation, diagnosis of generalized pustular psoriasis (GPP) poses significant challenges for patients and physicians.
Due to the rarity of generalized pustular psoriasis, cases often go unrecognized by physicians who are not dermatologists.
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.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Ultragenyx CEO Dr. Emil D. Kakkis said federal regulators should focus more on biomarkers and less on clinical endpoints when it comes to drug approvals.
When 2 friends set out along Spain’s 500-mile Camino de Santiago, they didn’t know how many people they’d inspire with 3 little words: “I’ll Push You.”