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Edmonton, Canada –  A cross-border collaboration between researchers at the University of Alberta and Cornell University has successfully created a device which, once implanted under the skin, can secrete insulin without triggering an immune response. In a paper published in the journal Nature Biomedical Engineering, the scientists, led by James...
Amsterdam, Netherlands – The drug vamorolone (Agamree®) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). It has recently been approved for clinical use in the United States by the Food and Drug Administration (FDA) and in the EU by the European Medicines Agency (EMA)....
Rockville, MD – According to several key opinion leaders (KOLs) interviewed by GlobalData, pipeline gene therapies hold the greatest potential to transform the Gaucher disease landscape. While the importance of these novel therapies has been emphasised, there remains a significant amount of groundwork to be covered before these therapies receive...
An expert panel failed to reach consensus on how to treat some patients with severe hemophilia A, suggesting that further study is needed where the optimal treatment remains equivocal, according to research published in the Journal of Blood Medicine. Hemophilia A is linked with a reduced quality of life (QoL)...
New York, New York – Because bleeding from the reproductive tract is a naturally occurring event during menstruation and childbirth, women who exhibit menorrhagia, or excessive bleeding after their menstrual cycle, may have underlying diseases that are underdiagnosed. In order to address important issues related to the diagnosis and management...
Extended induction chemotherapy with topotecan, cyclophosphamide, and etoposide does not improve event-free survival (EFS) and overall survival (OS) in patients with high-risk neuroblastoma, and it increases the number of toxic side effects, according to findings published in the Annals of Oncology. The open-label, prospective, randomized controlled phase 3 NB2004-HR trial (ClinicalTrials.gov...