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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. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.

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To Share or Not to Share Failed Genetic Screening Results with Patients

Worldwide Clinical Trials

When using genetic screening to identify clinical trial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article!

Genetics 130
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Partnership to advance personalised treatments for long Covid

Drug Discovery World

The partners will first develop and validate genotypic diagnostics that provide clinicians with an accurate view of a patient’s risk of disease; then, using the molecular profile of their underlying disease mechanisms, report back effective prescribing decisions and drug discovery opportunities to improve patient outcomes.

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Large-Scale Study Shows Guardant360 Liquid Biopsy Accelerates Clinical Trial Enrollment Compared to Tissue Biopsy

BioTech 365

Nature Medicine Publication Reports that Liquid Biopsy More Than Doubles Clinical Trial Enrollment Rate Compared to Tissue Biopsy REDWOOD CITY, Calif.–(BUSINESS Despite advances in precision oncology, progress is slowed by the limitations of tissue genotyping, … Continue reading → Nasdaq: GH).

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. Terence Eagleton, MB BS, Senior Medical Director at the global clinical research organization (CRO) Medpace. Reference: Chung DC, et al.

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EU OKs paediatric use of Gilead's Epclusa

The Pharma Data

The decision allows use of the therapy in the EU in children as young as six years of age and weighing at least 17kg, regardless of HCV genotype or liver disease severity. Source link.

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Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002

BioTech 365

ETD002 is a novel TMEM16A chloride channel potentiator Therapy applicable to all cystic fibrosis patients, independent of genotype BRIGHTON, England–(BUSINESS WIRE)–Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives … Continue reading (..)