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.
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 European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted… read more.
Neurocrine Biosciences, Inc. announced the FDA approved Crenessity (crinecerfont) capsules and oral solution as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients… 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.
PTC Therapeutics, Inc. announced the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that… read more.
Positive high-level results of KOMET, the largest, global randomised double-blind placebo-controlled multicentre Phase III trial in adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas… 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.
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