2016

Shire’s phase III VONVENDI study meets primary endpoint

Monday, December 5, 2016

Shire, a biotechnology company focused on serving individuals with rare diseases and other highly specialized conditions, has announced topline results from a phase III clinical trial of VONVENDI [von Willebrand factor (Recombinant)] to treat bleeds in elective surgical settings for adults with severe von Willebrand disease (VWD), the most common inherited bleeding disorder.

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Report: Vast swathes of human biology untouched by drug discovery programs

Monday, December 5, 2016

Scientists have created a map of all 1,578 licensed drugs and their mechanisms of action—as a means of identifying ‘uncharted waters’ in the search for future treatments. Their analysis of drugs licensed through the FDA reveals that 667 separate proteins in the human body have had drugs developed against them—just an estimated 3.5% of the 20,000 human proteins.

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bluebird bio hires Susanna High and Andrew Obenshain

Monday, December 5, 2016

bluebird bio, a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, has announced two new appointments as part of the company’s ongoing preparation for commercial readiness. Susanna High was named chief operating officer and Andrew Obenshain was named senior vice president and head of Europe.

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Experience and compromise make all the difference

Monday, December 5, 2016

The Pulse on Study Conduct by Elizabeth Weeks-Rowe

It is the responsibility of the investigator to delegate to appropriately experienced staff. The scope of practice is defined by education, training and licensure. The investigator must recognize the capabilities and limits of staff to whom he/she has delegated study tasks, and assign accordingly.

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Report: Clinical research benefits the U.K. economy

Friday, December 2, 2016

An independent report, commissioned by the National Institute for Health Research (NIHR) Clinical Research Network and produced by KPMG’s Economics team, provides an assessment of the economic impact of the NIHR Clinical Research Network’s activities to support clinical research in the U.K. The report includes assessment of the economic contribution made to the U.K. economy through clinical research activity supported by the network; analysis to quantify the value of payments made to NHS Trusts for commercial studies, and the value to the NHS of pharmaceuticals used as part of industry-sponsored studies.

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Illumina launches iHope Program for pediatric undiagnosed genetic diseases

Friday, December 2, 2016

Illumina has announced the iHope program, a philanthropic initiative aimed at identifying the genetic causes of undiagnosed rare diseases in children. With initial program partners Foundation for the Children of the CaliforniasRare Genomics Institute and UCSF Benioff Children’s Hospital San Francisco, selected patients in financial need will receive clinical whole-genome sequencing performed by the Illumina Clinical Services Laboratory. Through whole-genome sequencing—the process of determining the order of all the DNA in a person’s body—Illumina and the iHope program partners strive to end years-long diagnostic odysseys of unnecessary and inconclusive testing for these children and their families.

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