Madrid, Spain – A few years ago, a patient was cured of multiple myeloma after being treated for hepatitis C, astounding researchers from the group led by Joaquín Martínez, from the H12O-CNIO Haematological Tumours Clinical Research Unit, a collaboration between the Hospital 12 de Octubre (H12O) and the National Cancer...
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Washington, DC – Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center for Biologics...
Basel, Switzerland – Some pathogens hide inside human cells to enhance their survival. Researchers at the University of Basel, have uncovered a unique tactic certain bacteria use to spread in the body without being detected by the immune system. In their study, they reveal the crucial role of a bacterial...
TOKYO, Japan — Astellas Pharma Inc. President and CEO Naoki Okamura contributed to the development of a pediatric formulation to treat schistosomiasis as a member of the Pediatric Praziquantel Consortium. The Consortium announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted...
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...