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Johns Hopkins brain scientists have figured out why a faulty protein accumulates in cells everywhere in the bodies of people with Huntington’s disease (HD), but only kills cells in the part of the brain that controls movement, causing negligible damage to tissues elsewhere. The answer, reported this week in Science,...
COPENHAGEN, Denmark — Genmab A/S (Nasdaq: GMAB) and Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) seeking to convert the accelerated approval of TIVDAK® (tisotumab vedotin-tftv) to full approval, for the treatment of patients with recurrent or metastatic...
NEW YORK, NY — Tiziana Life Sciences, Ltd. (Nasdaq: TLSA), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced it has submitted an FDA request to obtain Orphan Drug Designation for intranasal foralumab for the treatment of non-active secondary progressive Multiple Sclerosis (na-SPMS). This...
ANDERSON — “Every day is so different,” said Amanda Pfeiffer of Anderson. Pfeiffer was referring to her infant son, Kohen, who lives with a condition known as epidermolysis bullosa. “Like right now, he can’t be out in the heat and humidity, because it makes him blister really easily. It’s an...
Little Emma Gardner will have to take daily medication for the rest of her life after being born with a deadly genetic condition. Two-year-old Emma was diagnosed when she was just eight-weeks-old with cystic fibrosis, which affects her breathing and digestive system. Sufferers on average live only until their 30s...
Tokyo, Japan – Researchers from Tokyo Metropolitan University have identified how proteins collect abnormally in neurons, a feature of neurodegenerative diseases like Alzheimer’s. They used fruit flies to show that depletion of mitochondria in axons can directly lead to protein accumulation. At the same time, significantly high amounts of a...