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A Reflection on Shared Moments of Strength and Progress in the Rare Disease Communities

Conversations in Drug Development Trends

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

Disease 52
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Gianluca Pirozzi, MD, PhD, Celebrated for Dedication to the Field of Immunology and Rare Disease Research

The Pharma Data

Spending several years with Sanofi in Paris, France as a Global safety officer, he then relocated to New Jersey, USA to work in Early and Late Stage Clinical Development in Immunology, when he became the Global Project Head of Dupixent, a medicine that has revolutionized the field of allergic and atopic diseases.

Disease 52
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Foxp3 protein governs regulatory T cell genome structure

Drug Target Review

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. Nature reviews immunology [Internet] 2008 July [2023 November 10]; 8:523-32. The study was published in Nature Communications.

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Advanced 3D cell-based technologies

Drug Target Review

The chips can be modelled with primary cells, IPS derived stem cells or patient derived cells and potentiates to unlock molecular mechanisms that drive the disease pathophysiology, holding a greater promise. The first successful chip adaptation to a lung model was first described in 2010 by Donald Ingber, a bioengineer at Wyss institute.

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TEST: The Stanley Center for Psychiatric Research - History

Broad Institute

Thanks to the generous funding from Ted and Vada Stanley, and the collaborative environment of the Broad Institute, researchers at the Stanley Center have made strides toward understanding severe psychiatric disease with the goal of contributing to new understandings of pathogenesis, the identification of biomarkers, and above all, new treatments.

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Human neuronal cells: possibilities in drug safety testing

Drug Target Review

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 2008 Mar;153 Suppl 2(Suppl 2):S1-209. Using stem cell-derived neurons in drug screening for neurological diseases. Guide to Receptors and Channels (GRAC), 3rd edition.

Drugs 112
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Precision Medicine in the Genomic Era

DrugBank

Out of the millions of genetic variants present in human populations, GWAS are designed to uncover those associated with specific traits or diseases. 9 Polygenic diseases are much more common, and thousands of genetic variants with very small effects could impact their phenotype.

Disease 98