NICE (UK): 1.1 Eplontersen is recommended, within its marketing authorisation, as an option for treating hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy. It… read more.
Ionis Pharmaceuticals, Inc. announced that the FDA has approved Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare,… read more.
Merck Inc. (known as MSD outside of the United States and Canada), announced that the National Medical Products Administration (NMPA) in China has approved Welireg (belzutifan), for the… read more.
One treatment with a CRISPR-Cas9 based gene editing therapy, NTLA-2002, could replace daily medication of many patients with hereditary angioedema (HAE), a painful condition with severe onset of… read more.
A pharmacy professor at Binghamton University, State University of New York has a $1.9 million grant from the National Institute of General Medical Sciences to tackle gene therapy challengs…. read more.
Among things many families don’t wish to pass down to their children and grandchildren: medical issues. One in five parents say their child has been diagnosed with a… read more.
Chronic cough is among the most common reasons for seeking medical care, with middle-aged women the group most affected. New studies at Uppsala University also show that this… read more.
Scientists have identified a gene which, when missing or impaired, can cause obesity, behavioural problems and, in mothers, postnatal depression. The discovery, reported today in Cell, may have wider… read more.
Scientists have discovered genetic clues to the cause of restless leg syndrome, a condition common among older adults. The discovery could help identify those individuals at greatest risk… read more.
A new study in Heart Rhythm, the official journal of the Heart Rhythm Society, the Cardiac Electrophysiology Society, and the Pediatric & Congenital Electrophysiology Society, published by Elsevier, examined… read more.
– Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the FDA has… read more.
Vanderbilt University Medical Center received a $13 million Department of Defense grant to lead a multisite clinical trial that will evaluate repurposed FDA-approved drugs as treatment options for… read more.
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