Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and...
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Liam Little has a rare, life-threatening disease but even at his sickest, his mum Natasha says: “You couldn’t wipe the smile off his face.” The toddler, who has spent much of his life in hospital, was diagnosed with auto-immune enteropathy at nine months of age, a condition in which the...
BRONX, NY—Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)—two related blood diseases that disproportionally strike older adults—are notoriously difficult to treat and associated with high relapse rates. Although new therapies have improved survival, treatment options remain limited, and the prognosis for the 50% of people who experience disease relapse remains...
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...
EAST LANSING, Mich. – Researchers at Michigan State University have teamed up to create a new way to image immune cells in living subjects that could enable new treatment opportunities and better outcomes for patients with Type 1 diabetes. In particular, the new approach could help bolster the success of...
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...