Genetic test predicts obesity in childhood
What if we could prevent people from developing obesity? The World Obesity Federation expects more than half the global population to develop overweight or obesity by 2035. However,… read more.
What if we could prevent people from developing obesity? The World Obesity Federation expects more than half the global population to develop overweight or obesity by 2035. However,… read more.
The Simons Foundation Autism Research Initiative (SFARI) has released phenotypic and genetic data from the Autism Inpatient Collection (AIC), a cohort of more than 1,500 youth participants ages 4 to… read more.
Soleno Therapeutics, Inc, a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced one oral and six poster presentations at the 2025 United In… read more.
In a report recently published in the American Journal of Health-System Pharmacy, the Ochsner Health system delves into the multifaceted implementation of pharmacogenomics—a rapidly evolving field that examines how… read more.
NICE (UK): NICE is unable to make a recommendation on omaveloxolone (Skyclarys) for treating Friedreich’s ataxia in people 16 years and over. This is because Biogen withdrew its… read more.
A series of digital tests, carried out via a smartphone app, could enhance the detection of disease progression in Huntington’s disease and improve the efficiency of clinical trials,… read more.
An international team of researchers led by King’s College London have identified genetic factors that may make some individuals more or less sensitive to the environments they experience…. read more.
NICE (UK): Leniolisib is recommended, within its marketing authorisation, for treating activated phosphoinositide 3‑kinase delta syndrome (APDS) in people 12 years and over. Leniolisib is recommended only if the… read more.
A case in which a sperm donor was later found to be carrying a cancer-causing pathogenic variant in his gametes has highlighted the problems of regulating gamete donation… read more.
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.