Munich, Germany – MorphoSys is moving ahead with its investigational BET inhibitor pelabresib, a Phase III myelofibrosis drug candidate, even though it failed to meet one of two key endpoints in a recent trial. The biopharma’s decision to stick with pelabresib follows similarly mixed results for AbbVie’s myelofibrosis drug navitoclax...
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Eight-year-old Caleb Baird longs to feel like an average kid. To find that normalcy, he and his mother, Stephanie Frederick of Rochester, will go to Florida to be in the company of others who know exactly what they are going through. Three years ago, Caleb was diagnosed with common variable...
A petition calling for more research about a rare disease is to be presented to the Prime Minister next week. Bolton mother Susan Jones, whose daughter Emma Matheson died from sarcoidosis, has backed the campaign. Emma died in November last year aged 22 after a life-long battle with the disease...
SACRAMENTO, Calif. — For Mitch Ball, breakfast is a dizzying array of medication and cans of high-protein liquid formula, poured or pumped into his stomach through a tube. Afterward comes a 20-minute breathing treatment, then the grooming and the cleaning of the tubes and other circuits that keep the 24-year-old...
Two mothers who forged a friendship while caring for their severely disabled daughters are raising money for a “lifeline” children’s hospice. Jane Byrne’s daughter Sophie, seven, and Barbara Smyth’s 14-year-old girl Freya both suffer from multiple seizures every day caused by severe, disabling illnesses. But for both mothers, the trauma...
I have often said that we would try any treatment if it meant more mobility, strength, and health for our daughter Grace, who is living with Lambert-Eaton myasthenic syndrome (LEMS). Along with the various medicines and treatments approved by the U.S. Food and Drug Administration, some LEMS patients believe diet changes, vitamin...
BOSTON – Amyotrophic lateral sclerosis (ALS) is a severe, fatal neurodegenerative disorder causing loss of motor neurons and voluntary muscle action. While mouse studies have identified potential treatments, these drugs have typically done very poorly in human trials. Researchers at Boston Children’s Hospital, working in collaboration with Pfizer, now report...
New York, NY — Scientists at the Icahn School of Medicine at Mount Sinai have identified a new class of RNAs packed into tiny particles known as extracellular vesicles (EVs) that could revolutionize how cancer and other diseases are diagnosed. The team found that these molecules undergo changes when cancer...
New York, NY – Mount Sinai researchers have identified a key driver of a blood vessel disorder known as fibromuscular dysplasia (FMD) which affects up to five percent of the adult population and can lead to high blood pressure, heart attack, or stroke. In a study published in Nature Cardiovascular...
New York, NY – In a scientific breakthrough, Mount Sinai researchers have revealed the biological mechanisms by which a family of proteins known as histone deacetylases (HDACs) activate immune system cells linked to inflammatory bowel disease (IBD) and other inflammatory diseases. This discovery, reported in Proceedings of the National Academy...