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.
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.
Vertex Pharmaceuticals Incorporated announced that data on Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), also known in the European Union and in the U.K. as Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor,… 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.
PTC Therapeutics, Inc. announced that the European Commission (EC) has decided not to adopt the CHMP’s negative opinion of January 24, 2024 on the annual renewal of the… read more.
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