research News

San Francisco, CA – Children and adults with rare, deadly genetic diseases have fresh hope for curative therapies, thanks to a new collaboration between the Innovative Genomics Institute (IGI) and Danaher Corporation, a global life sciences and diagnostics innovator. The new Danaher-IGI Beacon for CRISPR Cures center will use genome...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor), which is approved in the United States and United Kingdom and is currently under review with health authorities in the EU, Canada, Australia, New Zealand and Switzerland. These data...
Philadelphia, PA – The new Kids First datasets advance our understanding of how genetics contribute to childhood cancers and congenital disorders, opening new doors for prevention and treatment. WHO: The Gabriella Miller Kids First Pediatric Research Program (Kids First), an initiative of the National Institutes of Health (NIH) WHAT: Kids...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from the first study on the impact of caring for a child with the ultra-rare genetic disorder, aromatic L-Amino acid decarboxylase deficiency (AADC-d). Results show that caregivers spend almost every waking moment caring for a child with AADC-d,...