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…
Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…
Happy Monday! This week, we have details on a sad story of a toddler passing away from very rare mesenchymal chondrosarcoma, new research about the genetic risk factors of non-alcoholic…
Happy Friday! This week, we have details on an investigational compound for beta thalassemia. Also, we break down the details of a new study that examines psychiatric illness rates among…
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