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Powell Center for Rare Disease Research and Therapy Department of Pediatrics in the College of Medicine
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  1. All Posts
  2. Pompe Disease
Posted in

Pompe Disease

Amicus Provides Updates on AT-GAA for Pompe Disease

January 7, 2020

DNA

The Assistance Fund Opens New Program for Pompe Disease

January 7, 2020

Family

Amicus Announces Positive Data in Phase 1/2 Study of AT-GAA at World Muscle Society

October 2, 2019

DNA

Amicus and Catalent Biologics Enter Partnership for Gene Therapy Development and Manufacturing

July 2, 2019

Partnership

Amicus and Brammer Bio Enter Gene Therapy Development and Manufacturing Collaboration

July 1, 2019

Partner

Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL in Pompe

December 20, 2018

Amicus Therapeutics

Accelerated Approval Denied & Study Underway for Pompe Treatment

September 10, 2018

Lab

UF & MDA Host 2018 Pompe Disease Patient Meeting

July 30, 2018

Healthy Life

Developing Personalized CRISPR-Cas9 Genome Editing Therapy for Pompe Disease

July 18, 2018

Lab

Amicus Announces Regulatory/Clinical Updates for AT-GAA in Pompe

July 6, 2018

Amicus

Recent News

  • Zolgensma-Evrysdi Combo Likely Beneficial for SMA Type 1 August 6, 2021
  • PepGen raises $113M to challenge Sarepta in DMD August 5, 2021
  • Sarepta Acquires Investigational Gene Therapy for LGMD2A August 4, 2021
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Powell Center for Rare Disease Research and Therapy Department of Pediatrics in the College of Medicine

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  • Pediatrics Department
  • Powell Gene Therapy Center
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Clinical and Translational Research Building
2004 Mowry Road, Gainesville, FL 32610
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