CymaBay, Inc. recently announced positive provisional results from its low-dose Phase II seladelpar clinical trial for primary biliary cholangitis (PBC) patients. Once you get past the confusing part of that…
The Associated Professional Sleep Societies held their 31st annual meeting in Boston June 3rd through 7th. You can read more about Sleep 2017 by clicking here. Among other discussions was cataplexy.…
More exciting news from the Annual American Society of Clinical Oncology Meeting last week! Onconova Therapeutics announced results from a Phase 2 study of oral rigosertib as a treatment for…
Attention hereditary angioedema patients: preventative treatment is on the horizon! Shire plc is one of the global leaders in rare disease research and pharmacology, and the group recently released positive…
Maya Doyle of Quinnipiac University and Allison Werner-Lin of the University of Pennsylvania conducted a scientific research study to understand the experience of adults and emerging adults living with cystinosis. There…
Vertex Pharmaceuticals announced that two phase 3 studies involving a combination of tezacaftor / ivacaftor improved the lung function in cystic fibrosis patients that are 12 and older, with the F508del…
Welcome to December 2015 PatientWorthians! 'Tis the season for sharing and do we have some stories for you! As we round #NERVEmber this week, we want to highlight some hope for our…
Happy Monday! This week, we have stories on a potential treatment approach for an aggressive form of mitochondrial disease, an experimental treatment for osteosarcoma earning Rare Pediatric Disease designation, and…
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