Washinton, DC – The FDA on Monday published a revised draft guidance document, aimed at helping sponsors and pharmaceutical companies to develop therapies for Alzheimer’s disease, particularly in its early stages before the onset of overt dementia. Monday’s draft document updates the FDA’s previous guidance, released in February 2018. The...
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Many people with Lambert-Eaton myasthenic syndrome (LEMS) wait an extremely long time for a diagnosis. Our family waited nine months, a relatively short time compared with others in the LEMS community. As we waited for a diagnosis to be confirmed for our 16-year-old daughter Grace, a fog of uncertainty hung over our...
NORTH BETHESDA, Md. — The Foundation for the National Institutes of Health (FNIH) announces the launch of a public-private partnership to build the largest data source for amyotrophic lateral sclerosis (ALS) and expedite the identification of biomarkers and clinical outcome assessments that would allow earlier diagnosis and accelerated drug development...
EAST LANSING, Michigan – Barth syndrome (BTHS) is an ultra-rare but debilitating genetic disease affecting cell mitochondria. High infant mortality due to cardiac complications and a weakened immune system significantly decrease life expectancy. Current medications treat symptoms but not the cause, leaving an unmet clinical need to develop effective treatments...
Tokyo, Japan – Leukemia is a common term used to refer to a form of blood cancer. However, there are different types of leukemia depending on the cell type involved. One unique form is myeloid/natural killer (NK) cell precursor acute leukemia (MNKPL). Because of its rarity, there is no consensus...
Toronto, Ontario – Using a novel methodology, researchers at The Hospital for Sick Children (SickKids) are the first in paediatric research to use data from an international real-world cohort to overcome the barriers associated with conducting randomized clinical trials in children with rare diseases. The gold standard for evaluating new...
NEW YORK, NY – The Human Immunome Project (HIP), a global nonprofit scientific initiative, released its Scientific Plan today, on World Immunology Day, the organization announced. The plan provides a detailed roadmap of how the Human Immunome Project and its network of global study sites will generate the world’s largest...
Bellingham, Washington – In the realm of biomedical sciences, the quest for accurate and efficient diagnostic tools is ever-evolving. One such promising innovation making waves is the photoacoustic (PA) technique. In the past decade, PA imaging has emerged as a viable imaging modality demonstrated in many clinical applications with promising...
SEOUL, South Korea and HYDERABAD, India — The International Vaccine Institute (IVI), an international organization with a mission to discover, develop, and deliver safe, effective, and affordable vaccines for global health, today announced that it has commenced a technology transfer of simplified Oral Cholera Vaccine (OCV-S) to Biological E. Limited...
BAR HARBOR, Maine – The Jackson Laboratory, an independent, nonprofit biomedical research institution, announced the appointment of Lon Cardon, Ph.D., FMedSci, a pioneer in human genetics and drug discovery, as its next president and chief executive officer. Effective on November 29, Cardon will succeed current President and CEO Edison Liu...