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Spinal Muscular Atrophy Drug Pipeline - 2012

Clinical Development. Preclinical: Discovery. Spinal Muscular Atrophy Drug Pipeline - 2012. FDA Approval. Basic Research Seed Ideas. Identification Optimization Safety & Manufacturing. Phase I Phase II Phase III. IND. Trophos/Olesxime. Repligen/Quinazoline*.

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Spinal Muscular Atrophy Drug Pipeline - 2012

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  1. Clinical Development Preclinical: Discovery Spinal Muscular Atrophy Drug Pipeline - 2012 FDA Approval Basic Research Seed Ideas Identification Optimization Safety & Manufacturing Phase I Phase II Phase III IND Trophos/Olesxime Repligen/Quinazoline* ISIS - Biogen/Antisense* CSC/Motor Neuron* OSU/Gene Therapy* NINDS/Indoprofen Paratek/Tetracycline* *Projects with FSMA FundingInvolvement PTC - Roche/SMN Regulators Novartis/SMN Regulators Repurposed Drugs Studied for SMA Valproic Acid* Riluzole Phenylbutyrate* Hydroxyurea Salbutamol CALiBR SMN Regulators NCGC - IU/SMN Regulators NINDS/Benzimidazoles Harvard /SMN Regulators IU = Indiana University NCGC = NIH Chemical Genomics Center NINDS = National Institute of Neurological Disorders and Stroke

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