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CAMBRIDGE & NOTTINGHAM, England – Exonate Ltd., a biotechnology company developing novel, non-invasive, small-molecule therapeutics for patients with retinal vascular diseases, today announced the data from a successful Phase Ib/IIa trial for lead candidate EXN407. These data demonstrate the safety and tolerability of EXN407, as well as clear indications of...
A man who is unable to move or speak can now generate words and sentences on a computer using only his thoughts. The ability comes from an experimental implanted device that decodes signals in the man’s brain that once controlled his vocal tract, as researchers reported Wednesday in The New England Journal of...
Baltimore, Maryland – Combining a pair of experimental drugs may help treat malignant peripheral nerve sheath tumors with fewer harmful side effects, according to preliminary animal studies led by investigators at the Johns Hopkins Kimmel Cancer Center and Johns Hopkins Drug Discovery. The study, published Dec. 1 in the journal Molecular Cancer Therapeutics, shows that combining...
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...