Philadelphia, PA – Thursday afternoon marked a major regulatory milestone. In one of the year’s most highly anticipated FDA decisions, the regulator approved Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) as the first-ever therapy for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH). Or was the approval for metabolic dysfunction-associated steatohepatitis (MASH)? If...
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AUSTIN, Texas — Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, today announced the publication of a peer-reviewed manuscript in the Journal of Clinical Oncology – Precision Oncology (JCO PO). The paper features results from a multi-institutional study evaluating circulating tumor DNA (ctDNA) as a prognostic biomarker for patients...
DUBLIN – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the National Comprehensive Cancer Network® (NCCN) added Rylaze™ (asparaginase erwinia chrysanthemi (recombinant)-rywn) to the Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia (ALL), for both pediatric and adult patients. The NCCN Guidelines for ALL and the NCCN Guidelines for Pediatric...
Bethesda, Maryland – Scientists at the National Institutes of Health (NIH) and their colleagues have identified a gene responsible for some inherited retinal diseases (IRDs), which are a group of disorders that damage the eye’s light-sensing retina and threatens vision. Though IRDs affect more than 2 million people worldwide, each...
OXFORD, England – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the National Institute for Health and Care Excellence (NICE) has issued a positive Final Appraisal Document (FAD) recommending Sunosi®▼ (solriamfetol) for adults with excessive daytime sleepiness (EDS) caused by narcolepsy.[1] Solriamfetol is recommended as an option for treating EDS in...
North Bethesda, Maryland – Studies of interactions between two lab-generated monoclonal antibodies (mAbs) and an essential Epstein-Barr virus (EBV) protein have uncovered targets that could be exploited in designing treatments and vaccines for this extremely common virus. The research was led by Jeffrey I. Cohen, M.D., and colleagues from the...
MINNEAPOLIS — Every day, thousands of people with leukemia and other life-threatening diseases search the Be The Match Registry for a bone marrow match. To highlight the enormous need for more bone marrow donors, Be The Match(SM), the new name for the National Marrow Donor Program(R) Registry, kicks off its...
NORWELL, Mass. — The National Organization for Rare Disorders (NORD®) today announced the addition of seven leading academic medical and research centers to its national NORD® Rare Disease Centers of Excellence Network, expanding collaboration to improve care and accelerate research for the more than 30 million Americans living with rare conditions. With...
National Organization for Rare Disorders (NORD®) Founder and past President Abbey S. Meyers, PhD, former U.S. Rep. Jaime Herrera Beutler of Washington State, New York Yankees centerfielder Bernie Williams, and pioneer clinical geneticist, Dr. Ada Hamosh, headline a group of rare disease champions who will be honored for their outstanding...
Singapore – A research team from the National University of Singapore (NUS) has unearthed new findings which may help explain the connection between cancer risk and poor diet, as well as common diseases like diabetes, which arise from poor diet. The insights gained from this study hold promise for advancing...