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Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.

Tahseen Mozaffar | Lionel Riou França | Jérôme Msihid | Pragya Shukla | Irina Proskorovsky | Tianyue Zhou | Magali Periquet | Kristina An Haack | Laurence Pollissard | Volker Straub
Molecular genetics and metabolism reports | 2024

The efficacy of avalglucosidase alfa (AVA) versus alglucosidase alfa (ALG) on forced vital capacity percent predicted (FVCpp) in patients with late-onset Pompe disease (LOPD) has been assessed in the Phase 3 COMET trial (NCT02782741). Due to the rarity of LOPD and thus small sample size in COMET, additional data were analyzed to gain further insights into the efficacy of AVA versus ALG.

Pubmed ID: 39035044

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Associated grants

  • Agency: NCATS NIH HHS, United States
    Id: UL1 TR001414

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RRID:SCR_018080

Independent, non-profit organization that has developed global data-sharing and analytics platform to promote, coordinate, and facilitate scientific sharing and reuse of clinical research data through creation and implementation of sustainable global data-sharing enterprise. Our focus is on sharing individual participant-level data from completed clinical trials. Users can search listed studies, request data sets from data contributors, aggregate data, or share data of their own. Vivli (Center for Clinical Research Data) is launching a portal to share participant-level data from COVID trials.

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