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 four years… 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.
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.
Octapharma USA, Inc. has announced the expanded approval of fibryga, Fibrinogen (Human) Lyophilized Powder for Reconstitution, for fibrinogen replacement in bleeding patients with acquired fibrinogen deficiency (AFD) by… read more.
Zai Lab Limited and argenx announced that China’s National Medical Products Administration (NMPA) approved the Biologics License Application (BLA) for efgartigimod alfa injection (subcutaneous injection) (efgartigimod SC), 1,000mg… read more.
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