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Inside Precision Medicine Small Molecule Shows Promise for Alternative Sickle Cell Disease Treatment

Inborn genetic diseases

Sickle Cell disease - 3D Illustration of sickled blood cells on a dark background
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Small Molecule Shows Promise for Alternative Sickle Cell Disease Treatment

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Red Blood Cells 3D Image Concept
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Pfizer Gene Therapy for Hemophilia A Nets Positive Results, but Leaves Questions

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Bone marrow illustration
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CRISPR Editing Strategy May Eliminate Toxic Conditioning for Cell Therapy

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Red Blood Cells 3D Image Concept
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CASGEVY Is Durable in over 100 Patients, Editas’ Reni-cel Shows Clinical Promise

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Motor neuron connecting to muscle fiber to illustrate myasthenia gravis
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Genetic Cause of Spinocerebellar Ataxia 4 Identified

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Sickle Cell disease - 3D Illustration of sickled blood cells on a dark background
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Can Gene Therapy Be Affordable? U.S. Tries New Access Model in Sickle Cell Disease

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Image showing heart failure
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BridgeBio Nabs About $1.25B to Power Genetic Medicine and Cancer Pipeline

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An illustration of a CRISPR-Cas9 gene editing protein cutting a DNA helix
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In Another Win for CRISPR, FDA Approves Casgevy for Beta Thalassemia

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Sickle Cell disease - 3D Illustration of sickled blood cells on a dark background
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Two Gene Therapies for Sickle Cell Disease Get FDA Nod, Including First Ever CRISPR-Based Therapy 

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CRISPR
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U.K. Approves Vertex/CRISPR Tx’s Casgevy for Sickle Cell and Beta Thalassemia

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