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...
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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,...
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...
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...
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)...
By Raffaele Pereno – PhD, MBA BASEL, Switzerland – Reporting from the Festival of Biologics 2025, CheckOrphan attended a thought-provoking keynote panel featuring Dr. Marco Traub, Founder of the Trans European Stem Cell Therapy Society (TESCT), and Dr. Pedro Cruz, Chief Technical Operations Officer at iBET – Instituto de Biologia...