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FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. 0 genotypes. The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-?0/?0

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

Worldwide Clinical Trials

The best course of action will depend on many variables, such as the disease or population being studied and the genotype/phenotype relationship, risk, and impact on care. Obtaining appropriate assistance to facilitate planning up front for the complexities involved will save sponsors time and effort in the long run.

Genetics 130
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Risk adjusted net present value: What is the current valuation of Pharma Mar’s Ecubectedin?

Pharmaceutical Technology

The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress. Pharma Mar offers in vitro diagnostic products used for detection of virus and bacteria in respiratory infections and human papilloma virus genotypes, among others.

Hormones 100
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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

For rare disease studies where there is little or no clinical trial experience, outcome assessments would have to be validated within a clinical development program. Early and ongoing engagement with regulators greatly helps clinical development planning.

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Global Roundup: Overland Pharma Launches in China to Provide Access to Breakthrough Therapies

The Pharma Data

The aim of the research will be to create a TGS IVD platform by optimizing a multi-layer approach encompassing the initial sample and library preparation, state-of-the-art sequencing technologies and improved genotyping procedures. Amolyt has also acquired an option to license the identified candidates for future clinical development.

In-Vivo 52
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Top 7 Pharma Industry Leaders in 2020 By the Numbers

Delveinsight

MAVYRET (Glecaprevir and pibrentasvir) (for Chronic HCV genotype), Skyrizi (Risankizumab) (for plaque psoriasis), and VENCLEXTA (Venetoclax) (for Chronic lymphocytic leukemia, and Acute Myeloid Leukemia) generated nearly same amount of revenue share i.e. 0.3% (USD 1.8 Imbruvica contributed USD 5.3 billion , around 11.5% of revenue share.

Vaccine 98
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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Visit OXLUMO.com for more information, including full Prescribing Information.