Eculizumab reduces relapse-related hospitalizations in patients with NMOSDFebruary 29, 2020Rare DiseasesMultiple Sclerosis
Study delineates spectrum of Dravet syndrome phenotypesDecember 10, 2019Epilepsy & SeizuresRare Diseases
Reduction in convulsive seizure frequency is associated with improved executive function in Dravet syndromeDecember 10, 2019Epilepsy & SeizuresRare Diseases
AVXS-101 may result in long-term motor improvements in SMANovember 18, 2019Rare DiseasesPediatricsNeuromuscular Disorders
Ataluren shows real-world benefit for nonsense mutation Duchenne muscular dystrophyNovember 7, 2019Neuromuscular DisordersRare Diseases
CMT1A neuropathy improves with investigational drug PXT3003October 29, 2019Neuromuscular DisordersRare Diseases
Baricitinib may benefit patients with Aicardi-Goutières syndromeOctober 28, 2019Rare DiseasesPediatrics
Neurologists publish consensus statement on stridor in MSAOctober 15, 2019Rare DiseasesSleep MedicineNeuromuscular Disorders
C-Path and NORD team up to speed development of treatments for rare disordersOctober 7, 2019Rare Diseases
‘Pot’ is still hot for Dravet, Lennox-GastautAugust 22, 2019Epilepsy & SeizuresRare DiseasesPediatrics
