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...
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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 – 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...
New York, NY — Recent developments in cancer research have highlighted the vital role of the immune system, particularly in the notable successes of cancer immunotherapy. Now, a paradigm-shifting study led by researchers at the Icahn School of Medicine at Mount Sinai in New York in collaboration with the University of...
The classic symptoms of Parkinson’s disease involve tremor, stiffness and slow movements. Over the last decade, neurologists have been paying greater attention to non-motor symptoms, such as digestive and sleep problems, loss of sense of smell and depression. A genetically engineered mouse reproduces many of the non-motor symptoms associated with...
Twenty-four parliamentarians across party lines have approached Union health and family welfare minister Mansukh Mandaviya, underlining the need for sustainable funding support for certain diseases under the National Policy for Rare Diseases 2021.