Rare diseases evolved from anomalies to recognized conditions, with key advances in genetics, treatment, and global initiatives.
Pediatric FD poses a complex clinical challenge, with early symptoms, progressive multiorgan involvement, and diagnostic delays. Early diagnosis and intervention are key.
A study shows that AI outperforms doctors in complex diagnoses. However, integrating it into medical practice requires more than just technology.
An article published in the NEJM presents new technologies that could restore communication in ALS: a breakthrough for paralysed patients.
The study used data from over three million children and focused on autism spectrum disorders, intellectual developmental delays, language development disorders and epilepsy.
Only a third of USA-approved oncology drugs seem to improve overall survival. This is the sobering conclusion of a retrospective analysis of US approvals.
The deficiency of the anion transporter PAT1 leads to a disrupted intestinal barrier, microbial dysbiosis and increased susceptibility to intestinal inflammation.
Rheumatoid arthritis treatment is often complex. If patients do not respond to therapy, the question arises as to which drugs can help.
62-year-old woman with fatigue, jaundice, fever, and kidney injury. Can you guess the diagnosis?
No available medical therapy has resulted in a reduction in mortality among patients with HFpEF. Therefore, the current aims of medical treatment are to reduce the risk of hospitalization and improve quality of life.
Dr. James Styner transformed trauma care after a 1976 plane crash, creating the ATLS protocol (now a global benchmark) to standardize emergency treatment.
Current guidelines recommend routine postoperative hormone treatment to control pain in endometriosis. But which treatment regimen is most effective?
A study shows that as a result of the “close parent intervention” program, FCC practices were improved in 8 NICUs.
A recent cost-effectiveness analysis addressed the question of the appropriate age for the start and end of screening colonoscopies.
Liver transplantation is a rapidly developing field. Advanced technologies, like AI, will improve access, outcomes, and offer more personalized care.
Tafasitamab plus lenalidomide and rituximab outperformed lenalidomide plus rituximab and a placebo in patients with relapsed or refractory follicular lymphoma.
Auto-SCT did not improve mantle cell lymphoma with undetectable minimal residual disease outcomes after induction therapy.
Daratumumab plus lenalidomide had superior PFS outcomes vis-a-vis lenalidomide plus dexamethasone in frail patients with untreated MM.
Allo-SCT was more efficacious than standard-of-care in paediatric patients with sickle cell anaemia and abnormal cerebral artery velocities.
For VTE patients who needed extended anticoagulation, a reduced DOAC dose did not meet non-inferiority criteria for efficacy, compared to full DOACs.