Spark Therapeutics: An encouraging attempt to dethrone enzyme replacement as the standard of care treatment for Pompe Disease.

On Saturday, October 6, 2018, Spark Therapeutics (ONCE) presented positive data from a preclinical trial with SPK-3006 for the treatment of Pompe disease in an oral presentation at the 23rd International Congress of the World Muscle Society in Argentina.

SPK-3006 is an investigational liver-directed adeno-associated viral (AAV) gene therapy being developed for Pompe disease.

Pompe disease is caused by the buildup of a complex sugar, glycogen, in the body’s cells. It is a rare, inherited, progressive and . . .

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