Several investigational therapies are being evaluated for the treatment of Prader-Willi syndrome, a rare disease marked by persistent hyperphagia.
Caregivers of patients with Prader-Willi syndrome said they need new treatments so much that they are willing to accept the potential risks they may carry.
The American College of Obstetricians and Gynecologists now recommends that all women—ideally before pregnancy—be screened for hemoglobinopathies.
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.