Geographic atrophy due to age-related macular degeneration (AMD) is the leading cause of irreversible blindness and affects more than 5 million persons worldwide. No therapies to restore vision in ...

Patients with muscle-invasive bladder cancer have varied outcomes after cystectomy. Circulating tumor DNA (ctDNA)–based detection of molecular residual disease may identify patients at high risk ...

In the phase 3 EMBARK trial, enzalutamide plus leuprolide and enzalutamide monotherapy were associated with longer metastasis ...

Patients with previously untreated, locally advanced, unresectable or metastatic triple-negative breast cancer who are not ...

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 ...

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 ...

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; ...

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 ...

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

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