Research on potential systemic sclerosis therapies is undergoing a remarkable transformation, propelled by advancements in innovative technologies.
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.
Hematologist Federico Stella, MD, says restrictive diets for patients in the hospital recovering from stem cell transplants are unnecessary.
Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.