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FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

It “could have been easily repurposed for DMD,” said Pier Lorenzo Puri at Sanford Burnham Prebys Medical Discovery Institute in San Diego, who has been working on the drug development since 2004. MDA supported development of a mouse model that enabled the preclinical studies of what would become Duvyzat.

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Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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Repurposing FDA-approved drugs may help combat COVID-19

The Pharma Data

Several FDA-approved drugs – including for type 2 diabetes, hepatitis C and HIV – significantly reduce the ability of the Delta variant of SARS-CoV-2 to replicate in human cells, according to new research led by scientists at Penn State. Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro.

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

The Pharma Data

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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Next Generation Sequencing (NGS) in Clinical Trials: Challenges and Opportunities

Vial

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

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Leveraging ADCs in precision oncology strategy

Drug Target Review

At a base-layer, Caris’ comprehensive molecular profiling that we perform for patients today can identify predictive biomarkers for current FDA-approved ADCs. Proximity labeling on tissue from patients that did not respond to current therapeutics may also help identify targetable proteins involved in drug resistance mechanisms.

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The Long Road to End Tuberculosis

Codon

As an undergraduate biology student, I spent some time in a TB lab working on antibiotic resistance — a growing concern for drug developers. Food and Drug Administration (FDA)-approved drug library, some of which inhibit E. The researchers were left with 40 drug candidates.

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