Regeneron Pharmaceuticals, Inc. announced that the FDA has approved Evkeeza (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of… read more.
NICE (UK) – Garadacimab can be used as an option to prevent recurrent attacks of hereditary angioedema in people 12 years and over, only if: i) they have… read more.
Mayo Clinic researchers have developed a genetic test that can help predict how people will respond to weight loss medications such as GLP-1s. The test estimates an individual’s… read more.
In a Policy Forum, Natalie Ram and colleagues discuss the concerning gaps in robust regulatory protection on direct-to-consumer genetic data and biospecimens. After declaring bankruptcy in March 2025,… read more.
Agios Pharmaceuticals Inc. announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) of Pyrukynd (mitapivat), an… read more.
Genetic testing of IVF-created embryos could help more women over 35 have a baby in less time, a clinical trial by researchers from King’s College London, King’s College… read more.
Ionis Pharmaceuticals,Inc. announced that the FDA has approved Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age… read more.
NICE (UK): Olipudase alfa is not recommended, within its marketing authorisation, for treating acid sphingomyelinase deficiency (ASMD; Niemann–Pick disease) in people with type AB or type B. This… read more.
Bayer announced that the FDA has approved Jivi, a recombinant DNA-derived, extended half-life factor VIII concentrate, for use in pediatric patients 7 years of age and older with… read more.
The EMA revealed that Pfizer had withdrawn its application submitted on December 20, 2024, for variation to the marketing authorisation for Ngenla to treat adults with growth hormone… read more.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.
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