Additional efforts are needed to improve quality of life and mitigate suffering in people with sickle cell disease. The field ...

The concept of adjuvant therapy for cancer was developed at a time when systemic cancer treatment was limited to cytotoxic chemotherapy that could only be administered intermittently but rarely ...

When a pediatrician caring for patients with a rare genetic disease discovers incidentally that he carries his own rare high-risk mutation, he must learn from a patient’s parents how to shift his ...

A baby boy was transferred to a tertiary care hospital 5 days after birth. He had an intact manubrium but no corpus of the sternum, which had resulted in complete displacement of the heart outside ...

A 43-year-old woman with bipolar disorder and advanced HIV infection was admitted to this hospital because of depressed mood, suicidal ideation, cough, and a temperature of 39.5°C. A diagnosis was ...

Digital tobacco-cessation treatments hold great promise. But dedicated efforts will be required to promote access to and ...

Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; ...

Monoclonal gammopathy of undetermined significance (MGUS) is a common premalignant plasma-cell proliferative disorder that is present in approximately 5% of the general population over the age of 50 ...

Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, multisystem, progressive, debilitating, and fatal disease characterized by tissue deposition of misfolded transthyretin ...

Bronchiectasis guidelines are inconsistent with regard to the effectiveness of mucoactive agents, and their use varies geographically. Large trials are needed to assess safety and effectiveness. For ...

We thank Sally Collins, M.Sc., M.A., for her help in researching and developing the glossary provided in the Supplementary Appendix, and Calvin Chou, M.D., Ph.D., and ...

Sotatercept, an activin-signaling inhibitor, reduces morbidity and mortality among patients with long-standing pulmonary arterial hypertension. Its effects in patients with pulmonary arterial ...