Vertex Pharmaceuticals announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic… read more.
BioMarin Pharmaceutical Inc. announced new data from studies of Voxzogo (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal… read more.
Mirum Pharmaceuticals, Inc. announced that the FDA has approved Ctexli (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Ctexli is the first… read more.
Certain common genetic changes might make some people with focal epilepsy less responsive to seizure medications, finds a new global study led by researchers at UCL and UTHealth… read more.
A new study from Queen Mary University of London found that 9% of all reported adverse drug reactions (ADRs) reported to the UK medicines regulator are associated with… read more.
Eton Pharmaceuticals Inc announced that it has launched Galzin (zinc acetate) capsules. Galzin is FDA-approved for the maintenance treatment of patients with Wilson disease who have been initially… read more.
Influencers are promoting “overwhelmingly” misleading information about medical tests on Instagram and TikTok, according to a global University of Sydney-led study published today in JAMA Network Open. Researchers analysed… read more.
Chenodal has received a medical necessity determination by the FDA for the treatment of cerebrotendinous xanthomatosis. Because of the potential hepatoxicity of Chenodal, poor response rate in some… read more.
Pharmacogenomics – an individual’s genetic response to medications – is an increasingly important strand of personalised healthcare but little is known about the public’s views on it. Researchers… read more.
Merck, known as MSD outside of the United States and Canada, announced that the European Commission (EC) has conditionally approved Welireg (belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α)… read more.
Ann & Robert H. Lurie Children’s Hospital of Chicago launched a newborn screening awareness campaign, with signage on public transit and billboards across Chicago urging parents to contact… read more.
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.
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