On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler When faced with your own mortality,…
by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…
Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
According to a recent press release, Japan just approved ULTOMIRIS (ravulizumab) for patients with atypical hemolytic uremic syndrome (aHUS). Developed by biopharmaceutical company Alexion Pharmaceuticals ("Alexion"), ULTOMIRIS represents a…
Recently, the European Medicines Agency (EMA) recommended Olumiant (baricitinib) for the treatment of adult patients with moderate to severe atopic dermatitis. Currently, Olumiant is approved for the treatment of…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a genetic disease that affects the central and peripheral nervous systems, as well as voluntary muscle…
According to a story from GlobeNewswire, Exagen, Inc. recently launched a testing panel called AVISE® Vasculitis AAV, which is designed to help physicians get fast and accurate data for patients…
For many years, researchers linked obesity to a number of other health conditions. Now, Taiwanese researchers identify a new cause for concern: worsening health outcomes for patients with ankylosing…
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