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,...
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Bethesda, MD – An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH). Currently, there is no treatment for...
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...
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...
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...
Los Angeles, Calif. – In one of the largest ever gene-environment interaction studies of red meat and colorectal cancer, which explored the impact of red meat consumption on a person’s cancer risk based on their genotype, researchers have identified two genetic markers that may help explain the association between the...
Norman, Oklahoma – People who are diagnosed with head and neck cancer often receive a standard type of chemotherapy as part of their treatment. If they are exposed to secondhand smoke during chemotherapy — even if they have never smoked themselves — the treatment may be far less effective at...