A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...

Fatty liver disease is not restricted to adults, and its incidence in children and adolescents is continuously increasing.

GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...

FDA approves 1st treatment for Menkes disease, offering a copper therapy that improves survival in affected children.

Hospitalizations for flu in children younger than age 18 have the highest weekly rate in more than 15 years. And in the ...

… but our independent journalism isn’t free to produce. Help us keep it this way with a tax-deductible donation today. Every year, thousands of parents hear the heart-wrenching news that their child ...

Please provide your email address to receive an email when new articles are posted on . The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ...

American kids are no longer routinely being recommended shots against hepatitis A, meningitis, rotavirus and the flu.

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

A University of Houston pharmacology researcher and expert in cardiac development has identified a new cause of—and repair ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

Chelsea Cirruzzo is a Washington correspondent at STAT, where she covers HHS. You can reach Chelsea on Signal at chelseacirruzzo.42. Helen Branswell covers issues broadly related to infectious ...