Birch bark derivative gel found effective for EB, in phase 3 studyNovember 3, 2020Rare DiseasesDermatology
Ataluren delays disease milestones in patients with nonsense mutation DMDOctober 27, 2020Rare DiseasesNeurology
JIA arthritis and uveitis flares ‘often run parallel’October 26, 2020Musculoskeletal DisordersRare Diseases
Nusinersen provides continued benefits to presymptomatic children with SMAOctober 22, 2020Rare DiseasesNeurologyMusculoskeletal Disorders
Golimumab approval extended to polyarticular-course JIA and juvenile PsAOctober 2, 2020Rare DiseasesMusculoskeletal Disorders
FDA adds polyarticular-course JIA to approved indications for tofacitinibSeptember 30, 2020Rare DiseasesMusculoskeletal Disorders
Canakinumab (Ilaris) tapering tested in systemic JIA trialAugust 19, 2020Rare DiseasesMusculoskeletal Disorders
FDA approves viltolarsen (Viltepso) for Duchenne muscular dystrophyAugust 13, 2020Rare DiseasesMusculoskeletal Disorders
First reported U.S. case of COVID-19 linked to Guillain-Barré syndromeJune 24, 2020COVID-19 UpdatesRare DiseasesNeurology
New research confirms the efficacy and safety of onasemnogene abeparvovec for SMAMay 19, 2020Rare Diseases
