Pediatrics
A 6-month-old girl was brought to the ED with a 3-day history of redness and swelling of the left fourth toe. A circumferential ring was seen constricting the middle phalanx of the toe.
Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival of 75 to 80%.
In this clinical trial of second-line antiretroviral therapy in children with HIV in Africa, regimens including tenofovir alafenamide fumarate as the backbone and dolutegravir as the anchor drug were most effective.
When a dying patient’s parents refuse to give up hope, her physician opts to hope with them, going to unusual lengths to provide a last treatment he knows will be futile.
1-20 of 4998 results
- In this clinical trial of second-line antiretroviral therapy in children with HIV in Africa, regimens including tenofovir alafenamide fumarate as the backbone and dolutegravir as the anchor drug were most effective.
- Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival of 75 to 80%.
- In this phase 3 trial involving participants with nonrelapsing secondary progressive multiple sclerosis, treatment with tolebrutinib resulted in a lower risk of disability progression than placebo.
- In this phase 2 randomized, placebo-controlled trial involving 112 participants with narcolepsy type 1, oveporexton significantly improved measures of wakefulness, sleepiness, and cataplexy over a period of 8 weeks.
- A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a urea-cycle disorder. The affected infant was treated at approximately 7 and 8 months of age.
- In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder.
- A 6-month-old girl was brought to the ED with a 3-day history of redness and swelling of the left fourth toe. A circumferential ring was seen constricting the middle phalanx of the toe.
- Musunuru and colleagues1 describe in the Journal an innovative approach in which gene editing is applied in the treatment of a very rare disease, the severe urea-cycle disorder carbamoyl-phosphate synthetase 1 (CPS1) deficiency, which is usually fatal in early infancy in approximately half the infants who...
- The dismantling of foreign-assistance efforts has already had, and will continue to have, severe effects on vulnerable populations globally. But it also has implications for people in the United States.
- When a dying patient’s parents refuse to give up hope, her physician opts to hope with them, going to unusual lengths to provide a last treatment he knows will be futile.
- This phase 1–2 study involving nine children with leukocyte adhesion deficiency type 1 showed significantly better survival with lentiviral gene therapy without hematopoietic stem-cell transplantation than that in historical controls.
- A pediatric oncologist’s outlook and practice are transformed when a 10-year-old cricket fanatic with acute lymphoblastic leukemia touches her heart.
- Unhealthy alcohol use is common and includes severe alcohol use disorder. Screening tests identify patients who may benefit from preventive counseling, medications, or more intensive intervention.
- A key function of leukocytes is their ability to migrate and extravasate into tissues, which is facilitated by cell surface molecules that promote adhesion to the vascular endothelium. The β2 integrin molecule (also known as CD18) is the common component of four molecular complexes that play a crucial...
- In this randomized trial conducted over the 2019–2024 influenza seasons, baloxavir resulted in a significantly lower incidence of transmission of influenza virus from patients to household contacts than placebo.
- A comparison of clinical outcomes in 39 patients treated with atidarsagene autotemcel, a hematopoietic stem-cell–based gene therapy, with those of untreated patients showed a strong treatment effect over the long term.
- In this randomized trial involving patients with noncystic fibrosis bronchiectasis, the rate of pulmonary exacerbations over a 52-week period was lower with brensocatib (10 mg or 25 mg per day) than with placebo.
- A 65-year-old man presented with 1-day history of a rash with a bull’s-eye appearance on his left hip. One week earlier, he had removed a tick from his hip after doing yard work near the woods by his home in Tennessee.
- The author describes the scientific foundations of a phase 3 study of brensocatib for the treatment of bronchiectasis.
- A 3-year-old boy presented with a 6-week history of ulcerated plaques with rolled borders and satellite papules on his left lower leg. He had recently immigrated from Venezuela after a long journey by land with his family.