3 Topic Commentaries

FDA approves therapy for GSDIa

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  Karim Mikhail, B. Pharm., M.S.

  Regulatory science; biologics and gene therapy

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  U.S. Food and Drug Administration (FDA)

  Today’s approval is a great milestone in using a gene therapy to treat this disease and improve the quality of life for people with this condition.

  Source
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  Megha Kaushal, M.D., MSc.

  Hematology/clinical evaluation of cell and gene therapies

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  U.S. Food and Drug Administration (FDA)

  This accelerated approval reflects our confidence in the clinical evidence to date and our commitment to bringing innovative treatments to patients with rare genetic diseases while we continue to gather data to confirm long-term benefit.

  Source
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  Dwight D. Koeberl, MD, PhD

  Medical genetics and metabolic disorders

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  Duke University School of Medicine

  A clinical trial of gene therapy for GSD Ia enrolled kids as young as eight and have seen lasting results at that age.

  Source