Cambridge, England – Voydeya (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular haemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris...
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WILMINGTON, Del. — AstraZeneca Pharmaceuticals LP and Ionis’ WAINUA™ (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered...
Farzana Nisha was about to lose her patience. She could not wait for the three-hour heart operation to end so she could see her two-year-old son. Fear overcame her after three hours and there seemed to be no end in sight to the surgery. Her son Rishab Prakash had a...
Chloe Bush is an adorable two year old that I visit and read to through a non-profit here in town called Circle of Hope. She has a terminal disease that is the #1 genetic killer of children under two. Chloe has surpassed that scary birthday but is still terribly ill...
Melbourne, Australia – Researchers from Melbourne-based medical research institute, WEHI, have unravelled how mutations in a gene can lead to an incurable neurodevelopmental disorder that causes abnormal brain development in newborns and infants. The study is the first to prove that a protein called Trabid helps control neuronal development, and that mutations...
SAN DIEGO, Calif. – Accelerated aging was more common in recent birth cohorts and was associated with increased incidence of early-onset solid tumors, according to research presented at the American Association for Cancer Research (AACR) Annual Meeting 2024, held April 5-10. “Multiple cancer types are becoming increasingly common among younger...
St. Louis, MO – Nearly all adults with Down syndrome will develop evidence of Alzheimer’s disease by late middle age. A new study by researchers at Washington University School of Medicine in St. Louis shows that the disease both starts earlier and moves faster in people with Down syndrome, a...
New Brunswick, N.J. – “Taking out the trash” takes on a whole new meaning, as investigators at The Cancer Institute of New Jersey (CINJ) and Rutgers, The State University of New Jersey, have discovered that a waste disposal protein is the key to cancer tumor suppression in a process known...
WATERTOWN, N.Y. (WWNY) – She may be small, but she sure is mighty! Madyson Siddall, from Watertown, is about to turn ten. She loves pranks, dogs, and stuffed animals. But Madyson has had a tough year. In February 2020, her legs began hurting, and she was feeling tired all the...
DETROIT, Mich. — Wayne State University postdoctoral research fellows Patrick Monaghan, Ph.D., and Michael VanNostrand, Ph.D., along with Nora E. Fritz, Ph.D., PT, DPT, NCS, director of the Neuroimaging and Neurorehabilitation Lab and associate professor of physical therapy in the Department of Health Care Sciences in WSU’s Eugene Applebaum College...