NCATS explores how best to use diagnostic codes to move field forward
treatment News
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...
SPRING, Texas — Io Therapeutics, Inc., a privately held pharmaceutical company headquartered in Spring, Texas; announced today publication online in Scientific Reports – Nature, of collaborative studies with Duke University scientists on effects of the company’s anti-cancer compound IRX4204, on human multiple myeloma, a fatal form of bone marrow cancer. The studies...
Montreal, Canada – A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. McGill University researchers have discovered that an experimental compound called K884 can boost the natural repair abilities of muscle stem cells. Current treatments can slow muscle damage,...
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...
HEFEI, China – A drug originally developed to treat myasthenia gravis has shown striking benefits in a patient with a rare and often debilitating neurological condition, offering new hope for people with limited treatment options. Doctors at Huashan Hospital have reported the first known case of successful treatment of Lambert-Eaton...
Washington, DC – A promising class of drugs called BTK inhibitors, already approved to treat certain cancers and currently in Phase III clinical trials at four different pharmaceutical companies for multiple sclerosis, has experienced multiple clinical holds and other stumbling blocks during the past two years. Yet physicians and researchers still...
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...