Pediatric NewsNORD Rare Disease Centers of Excellence: A new network seeks to break down barriers in rare disease careOctober 15, 2022Rare Diseases
Pediatric NewsThe urgent need to diagnose Sanfilippo syndrome at an early ageOctober 15, 2022Rare DiseasesNeurology
Pediatric NewsNovel gene-based therapies for neuromuscular diseasesOctober 15, 2022Rare DiseasesNeurology
Pediatric NewsWith sleuth work, pediatricians can identify genetic disordersOctober 12, 2022Rare DiseasesNeonatal Medicine
Pediatric NewsIncreasing primary care doctors’ knowledge of IPF could speed up diagnoses, suggests white paperSeptember 28, 2022PulmonologyRare DiseasesBusiness of Medicine
Pediatric NewsShift in child hospice care is a lifeline for parents seeking a measure of comfort and hopeSeptember 22, 2022Rare Diseases
Pediatric NewsUncombable hair syndrome: One gene, variants responsible for many casesSeptember 20, 2022DermatologyRare Diseases
Pediatric NewsAAP guidance helps distinguish bleeding disorders from abuseSeptember 19, 2022HematologyRare Diseases
Pediatric NewsKetamine promising for rare condition linked to autismSeptember 13, 2022NeurologyRare Diseases
Pediatric NewsCDC warns of enterovirus strain linked to polio-like conditionSeptember 13, 2022Rare DiseasesPulmonologyInfectious Diseases
Pediatric NewsFDA approves first gene therapy, betibeglogene autotemcel (Zynteglo), for beta-thalassemiaAugust 17, 2022HematologyRare Diseases
Pediatric NewsBiosimilar-to-biosimilar switches deemed safe and effective, systematic review revealsAugust 15, 2022GastroenterologyMusculoskeletal DisordersRare DiseasesDermatology
Pediatric NewsTwo deaths from liver failure linked to spinal muscular atrophy drugAugust 12, 2022Rare DiseasesNeurology
Pediatric NewsStopping JIA drugs? Many can regain control after a flareAugust 10, 2022Rare DiseasesMusculoskeletal Disorders
