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...
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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...
BALTIMORE – MyMD Pharmaceuticals, Inc. (Nasdaq: MYMD) (“MyMD” or “the Company”), a clinical stage pharmaceutical company committed to extending healthy lifespan by focusing on developing two therapeutic platforms, today announced that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,096,933 titled “Method of Treating Disorders...
BALTIMORE – Akers Biosciences, Inc. (Nasdaq: AKER), with its proposed merger partner MyMD Pharmaceuticals, Inc. (“MyMD”), a clinical stage pharmaceutical company committed to extending healthy lifespan by focusing on developing two therapeutic platforms, today announced new data from a study conducted by Eurofins Discovery Phenotypic Center of Excellence. MYMD-1 is...
NEW YORK, NY — Myrtelle Inc., a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation to the Company’s lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of...
CULVER CITY, Calif. – ImmunityBio, Inc (NASDAQ: IBRX), a clinical-stage immunotherapy company, today announced data from a Phase 1 pilot study showed N-803 combined with natural killer cells could have the potential to reduce viral load in people living with HIV. Published in The Journal of Infectious Diseases, researchers at...
SAN DIEGO, Calif. — n-Lorem, a nonprofit foundation, has announced the dates for its second annual Nano-rare Patient Colloquium, which will be held Oct. 30 & 31 in Cambridge, Mass. The Nano-rare Patient Colloquium will bring together a Community of Care for Nano-rare that consists of treating physicians, industry leaders,...
SAN DIEGO – n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, today announced a new partnership with Ultragenyx Pharmaceutical (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases. n-Lorem Foundation...