A study entitled “IER3IP1-mutations cause microcephaly by selective inhibition of ER-Golgi transport”, now published in the open access journal “Cellular and Molecular Life Sciences”, provides new insights into the molecular causes of a rare and usually fatal hereditary disease called MEDS1.
Updated: FDA approves Sarepta’s Duchenne muscular dystrophy gene therapy via accelerated pathway
The FDA granted accelerated approval to Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy for boys aged 4 and 5 in a decision that allows access
June 23, 2023