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    • Myotubular Myopathy
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Pompe Disease Category Subscribe to RSS Feed

Amicus Therapeutics

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

Published: Dec 20th, 2018

Lab

Accelerated Approval Denied & Study Underway for Pompe Treatment

Published: Sep 10th, 2018

Healthy Life

UF & MDA Host 2018 Pompe Disease Patient Meeting

Published: Jul 30th, 2018

Lab

Developing Personalized CRISPR-Cas9 Genome Editing Therapy for Pompe Disease

Published: Jul 18th, 2018

Amicus

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

Published: Jul 6th, 2018

Gene

Gene therapy for pulmonary dysfunction in Pompe

Published: Feb 19th, 2018

Valerion Initiates VAL-1221 Dosing in Patients with Pompe

Published: Jul 11th, 2017

The backstory behind the Pompe patient Trump highlighted to Congress

Published: Mar 2nd, 2017

The 2017 Rare Disease Day video!

Published: Jan 30th, 2017

Lab

UF researchers use gene therapy to treat pulmonary dysfunction in Pompe

Published: Sep 15th, 2016

Page 1 of 3123

Recent News

UF Health patient first in the nation to receive Zolgensma since approval

Jun 13th, 2019

Avexis Receives FDA Approval For Zolgensma

May 24th, 2019

The promise of gene therapy through collaboration: Pfizer’s view

May 24th, 2019

News Archive

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