Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Trump administration health officials assured drugmakers and researchers at a roundtable hosted by the FDA that they want to ...

Scientists have made a key breakthrough for treating respiratory diseases by developing a new drug delivery system that ...

DataM Intelligence | competitive Intelligence Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dys ...

Baby KJ Muldoon, the first patient to successfully receive personalized CRISPR gene editing therapy has returned home after over 300 days at the hospital.

Retinitis Pigmentosa therapies evolve beyond gene-specific fixes with gene-agnostic, intravitreal, and optogenetic advances ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

Newseria nie ponosi odpowiedzialności za treści oraz inne materiały (np. infografiki, zdjęcia) przekazywane w „Biurze Prasowym”, których autorami są zarejestrowani użytkownicy tacy jak agencje PR, ...

MODY 5 is a rare genetic diabetes subtype caused by HNF1B mutations or deletions, often presenting with early-onset diabetes ...

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...