Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions View by Topic View by Date Must Reads In Rare Diseases Rare Diseases for November/December 2020 Childhood Hodgkin survivors have neurocognitive impairment, Gene-replacement therapy shows promise in X-linked myotubular myopathy, More must reads Rare Diseases for September/October 2020 Ataluren delays disease milestones in patients with nonsense mutation DMD, Nusinersen provides continued benefits to presymptomatic children with SMA, CBD for LGS: Fewer seizures, but thrombocytopenia risk, More must readsRare Diseases for May/June 2020 Huntington's disease biomarkers appear 24 years before clinical symptoms, More must readsRare Diseases for January/February 2020 Inebilizumab benefits patients with NMOSD, Eculizumab reduces relapse-related hospitalizations in patients with NMOSD , Zilucoplan Improved Efficacy Outcomes in Myasthenia Gravis, Expedited Review Programs Not Shortening Drug Development, More must readsRare Diseases for November/December 2019 Study Delineates Spectrum of Dravet Syndrome Phenotypes, Reduction in Convulsive Seizure Frequency is Associated With Improved Executive Function in Dravet Syndrome, AVXS-101 May Result in Long-Term Motor Improvements in SMA, CMT1A Neuropathy Improves With Investigational Drug PXT3003, Ataluren shows real-world benefit for nonsense mutation Duchenne muscular dystrophy, More must readsPages1 2 3 4 last ยป
