Fragile X disorders include two distinct conditions: fragile X syndrome (the most common single-gene cause of developmental ...

This editorial explains the genetic basis of Pompe’s disease and other lysosomal storage disorders, as well as the foundations of enzyme-replacement therapy.

The U.S. Supreme Court recently upheld Tennessee’s ban on gender-affirming hormonal treatment for transgender minors. This decision will have profound implications.

The author describes the scientific foundations and implications of a study on variant EPO and erythrocytosis.

Among women with a history of early breast cancer treated with endocrine therapy (tamoxifen or aromatase inhibitors), vasomotor symptoms are common, occurring in up to 90% of this population, and o ...

The landscape for genetically modified organisms is changing, thanks to sharp increases in the amounts and numbers of chemical herbicides applied to GM crops and the classification of two of the mo ...

In 2006, a prospective observational study first showed that physical activity after a colon cancer diagnosis was linked to a decreased risk of recurrence and death.1 Since then, numerous observati ...

The Supreme Court recently agreed to hear a case about adolescent e-cigarette use and the FDA’s role in addressing it. A ruling against the FDA could have implications for its authority and for ...

The financial assistance policies of some U.S. hospitals now define medical necessity much more restrictively than it has conventionally been understood, as covering only services that are ...

A previously healthy 3-year-old boy presented with vomiting and fevers. Purpura developed on his body that evolved with the patient’s response to treatment.

At first glance, China might seem unlikely to offer useful health care lessons to other countries. But China has undertaken a series of remarkable health system experiments that are instructive at ...

A 4-year-old boy had a 2-day history of right hip pain and limping. A radiograph showed a focal area of collapse in the right femoral head.