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  14. Sarepta Therapeutics | Biopharmaceutical Company for Rare Diseases
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  18. Sarepta Therapeutics | Biopharmaceutical Company for Rare Diseases
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  26. Home | Bionomics – Clinical-stage biopharmaceutical company
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  30. Rare Disease and Orphan Drugs Journal
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  40. Glycomine Inc. - Developing therapies for critical unmet orphan diseases of glycosylation

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  80. Chimerix | Innovative medicines for cancer and other serious diseases | Home
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