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Proving chylomicrons matter

How Amsterdam Molecular plans to meet EMA's issues with LPL gene therapy

In asking EMA to reexamine an MAA for Glybera, Amsterdam Molecular Therapeutics Holding N.V. believes it will not need to enroll more patients to get the long-term efficacy data required by the agency to make the compound the first approved gene therapy outside of China.

In its June recommendation against approval of Glybera to treat lipoprotein lipase (LPL) deficiency, EMA focused on the efficacy of the adeno-associated virus (AAV)-based gene therapy, not on its safety, stating that the trial data "had not shown a

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