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Common genetic variants linked to drug-resistant epilepsy

Written by | 22 Apr 2025

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.

Testing patients for just three genes could help prevent three quarters of avoidable side effects of certain medications

Written by | 4 Apr 2025

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.

Chenodal has received a medical necessity determination by the FDA for the treatment of cerebrotendinous xanthomatosis – Mirum Therapeutics

Written by | 28 Feb 2025

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.

Almost 90% of people would agree to genetic testing to tailor medication use, survey finds

Written by | 25 Feb 2025

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.

Lurie Children’s campaign urges parents to follow up right away if newborn screening results are abnormal

Written by | 15 Feb 2025

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) positive for Wainzua (eplontersen) for treating hereditary transthyretin-related amyloidosis – AstraZeneca

Written by | 19 Jan 2025

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.

FDA approves Tryngolza (olezarsn) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome – Ionis Pharma

Written by | 9 Jan 2025

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.

NMPA (China) approves Welireg (belzutifan), for the treatment of adult patients with von Hippel-Lindau disease – Merck Inc

Written by | 17 Dec 2024

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.

Gene editing therapy shows promise for hereditary angioedema

Written by | 26 Oct 2024

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.

Pharmacy professor receives $1.9 million award to tackle gene therapy challenges

Written by | 15 Sep 2024

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.

2 in 3 parents want help preventing their child from developing hereditary health conditions

Written by | 31 Aug 2024

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 may be hereditary

Written by | 25 Aug 2024

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.

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