Vienna, Austria – The underlying cause of MS has not yet been fully clarified, but a connection with the Epstein-Barr virus (EBV) has long been suspected. In most patients who develop MS, specific immune responses against EBV are detectable, which are also directed against certain structures of the central nervous...
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ROCKLAND, Mass. and NEW YORK — The National Multiple Sclerosis (MS) Society, in collaboration with EMD Serono, today released findings from a nationwide GfK Roper survey, “MS Viewpoints: Understanding the Outlook on Emerging Therapies” in conjunction with the first-ever World MS Day. The MS Viewpoints survey compared the perspectives of...
Nashville, TN – Vanderbilt University Medical Center received a $13 million Department of Defense grant to lead a multisite clinical trial that will evaluate repurposed FDA-approved drugs as treatment options for patients with Rett syndrome. Affecting 1 in 10,000 females at birth, and males even more rarely, Rett syndrome is...
A new drug being studied for the treatment of muscle degenerating diseases has shown promising results. According to a study published today in the British Journal of Pharmacology, Debio 025 is as effective as current drugs but, crucially, does not cause unwanted immunosuppressive effects. Bethlem Myopathy and Ullrich Congenital Muscular...
WORCESTER, Mass. – Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the company has executed an exclusive license agreement with Leiden University...
WORCESTER, Mass. – Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has accepted the Company’s...
WORCESTER, Mass. — Mustang Bio, Inc. (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced Phase 1 clinical data were published in Nature Medicine that demonstrated the promising safety and...
What’s the connection between Gaucher disease, a rare single gene disorder of metabolism that appears during childhood, and Parkinson’s disease, a common multifactorial disorder of the nervous system that occurs late in life? The answer lies in just a single gene (glucocerebrosidase or GBA), which encodes an enzyme required for...
Dar es Salaam — The President of Zanzibar, Dr Hussein Mwinyi, has ordered state institutions to incorporate rare disease treatment in the health policy so that children facing such health issues can receive specialised care, particularly oxygen. Dr Mwinyi made the statement on Tuesday evening when he spoke to delegates...
The ground wobbles a little when your newborn is diagnosed with a rare disease. It threatens to swallow you whole when you learn a NICU attending has to distribute two stapled pages to each of his staff summarizing your daughter’s condition. They had never before treated a child with Hilde’s diagnosis, let alone...