Audentes Therapeutics is focused on developing and commercializing gene therapy products for patients suffering from serious, life-threatening rare diseases. CASQ2-related Catecholaminergic Polymorphic Ventricular Tachycardia. We believe patients, families and patient.

Gene and Cell Therapy Community Heatmap. National Gene and Cell Therapy Societies. Previous Congresses and Spring Schools. ESGCT offers a Young Investigator Award at the SETGyC meeting in Mallorca. Register for the SETGyC congress. Promotes basic and clinical research in. And regulatory bodies in Europe.

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We will send occasional emails announcing new publications, press releases, and events. Blue Ribbon Study Panel on Biodefense. Recommending and promoting changes to U. policy and law to strengthen national biodefense while optimizing resource investments.

You are not a member of this wiki. Regulatory, FDA, Expedited Pathways, Access, etc. Accelerating - Open Notebook Science. Audio and Video on Choroideremia Initiatives. Basic Research, Data, Registries. Cell Reprogramming, Optogenetics, etc. Clinical Trials and Pre-clinical Studies. CHM U Training Video Links. Welcome to the Choroideremia CHMINFO Wiki.

CLINICAL TRIAL OF GENE THERAPY FOR MPS VI. A SEVERE LYSOSOMAL STORAGE DISORDER. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. The results from this clinical trial proposed by the M.