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 ...

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; ...

Teclistamab, a T-cell engager targeting B-cell maturation antigen, showed promise as a rescue therapy in 10 patients with refractory autoimmune diseases and led to clinical and serologic improvemen ...

A 46-year-old woman presented with shortness of breath, dry cough, and painful rash on the upper arms. Examination was notable for erythematous lesions on the lateral upper arms and ventral surface ...

New models integrating clinic-based care with community-based services for people with substance use disorder could reduce the time to stable remission and support recovery.

Idiopathic intracranial hypertension, also called pseudotumor cerebri, refers to elevated cerebrospinal fluid (CSF) pressure that develops most often in women of reproductive age with obesity, causing ...

To the Editor: The Case Record discussed by Kotton et al. (June 19 issue)1 addresses a case of deceased organ donor–transmitted strongyloidiasis,1 a rare but serious complication of transplantation ...