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US FDA approves Vertex’s Orkambi for cystic fibrosis in children

Pharmaceutical Technology

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It enrolled 46 children aged one to under two years with the F/F genotype. According to the findings, Orkambi was found to be well tolerated.

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AMP recommends minimum set of pharmacogenetic alleles to guide clinical CYP2D6 genotype testing, pro

Scienmag

Latest joint consensus guideline authored by representatives from AMP, CAP, Royal Dutch Pharmacists Association, and European Society for Pharmacogenomics and Personalized Therapy ROCKVILLE, Md.

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Why early participant engagement is now a top priority in genetic disease research

pharmaphorum

Pharmaceutical companies and biotechs are also adapting their approaches, launching patient finding and engagement programmes that can start years before clinical trials begin and allow them to run ‘recontact by genotype’ studies that the Resilience Project would have liked to do. Giving participants something in return. About the author.

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Cancer, genomics and personalised medicine: Modelling the future of oncology

pharmaphorum

Personalised medicine in practice – where are we now in cancer? How the shift towards cancer as a phenotype/genotype is being applied in clinical trials and how they are run •What are co-clinical trials and why could they hold the key to answering unmet need in cancer treatment and beyond into broader drug development?

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Rare Disease Diagnosis: Why Tackling the Genomic Analysis Bottleneck is Key to Advancing Precision Medicine

XTalks

HS: Well, a definitive diagnosis opens the door to personalized medicine, potentially leading to reduced morbidity and mortality. It also slows the progress of identifying new disease gene associations in very rare conditions. What are the benefits of having a definitive diagnosis for patients with genetic disorders?

Genome 98
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The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

Worldwide Clinical Trials

They play a crucial role in tailoring hematology oncology therapeutic strategies to individual patients and have transformed personalized medicine in oncology. These recent advancements continue to broaden the pool of biomarkers, offering hope for improving patient outcomes via targeted therapies and precision medicine.

Genetics 195
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Inferring human genomes at a fraction of the cost promises to boost biomedical research

Scienmag

To discover these associations, researchers need to compare the genomes of many individuals at millions of genetic locations or markers, and therefore require cost-effective genotyping technologies. A new statistical method, developed […].

Genome 57