FDA OKs First ‘Targeted Treatment’ for Rare DMD Mutation

The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne muscular dystrophy (DMD) plus a rare DMD mutation, the agency has announced.  This particular mutation of the DMD gene “is amenable to exon 45 skipping,” the FDA noted in a press release. It added that […]

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Genomics, transcriptomics and proteomics shed light on autoimmune thyroid disease

Scientists at deCODE genetics, a subsidiary of Amgen, and their collaborators from the Icelandic healthcare system, University of Iceland and the Karolinska Institute in Sweden, have published a study in Nature comparing over 30,000 patients with autoimmune thyroid disease from Iceland and U.K. with 725,000 controls. Autoimmune thyroid disease (AITD) is the most common autoimmune disease and is highly heritable. […]

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Researchers uncover the genomics of health

Most diseases have a genetic component. To better understand disease, researchers led by the Garvan Institute of Medical Research are analysing genetic information to determine what keeps us healthy. In a world first, the team has compiled a genome reference database of thousands of healthy older Australians, which has the potential to predict disease-linked gene variants more accurately than has […]

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