The US president vowed to ‘end childhood cancer’. But his administration is dismantling the search for a cure and sending families scrambling for treatment ...

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations -- Dosing of the first participant in the Phase 1b ...

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant in the Phase 1b FALCON trial has been completed, with initial data expected ...

Much of pancreatic cancer’s evasive and deadly nature may come down to low levels of one gene. In A Nutshell Early detection, ...

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

Errors must be accepted as evidence of systems flaws, not character flaws.”—Lucian Leape1 In losing Lucian Leape to heart ...

The Global Medical Plastics Market Size projected to grow at a CAGR of 6.60% from 2026 to 2032, according to a new report published by Verified Market Research®. The report reveals that the market was ...

The cost to reach higher readiness levels is modest — an estimated $4–$48 per pediatric patient — with potential to ...

Caring for children with concussions can be daunting for pediatricians because these visits usually involve checking mental status and neurologic exams, said Corinna Rea, MD, MPH, pediatrician at ...

Caplacizumab is a von Willebrand factor (vWF)-directed antibody fragment designed to inhibit the interaction between vWF and platelets.

Systematic review finds convection-enhanced delivery in pediatric patients is linked mainly to low-grade adverse events, with no Grade 4 or 5 toxicity, supporting safer investigational CNS therapy ...

New nanomaterial passes the blood-brain barrier to reduce damaging inflammation after the most common form of stroke. When ...