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Amicus Therapeutics Concludes Pre-BLA Meeting With FDA For AT-GAA In Pompe  Disease
Amicus Therapeutics Concludes Pre-BLA Meeting With FDA For AT-GAA In Pompe Disease

Pompe Disease » Powell Center for Rare Disease Research and Therapy »  College of Medicine » University of Florida
Pompe Disease » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida

SEC Filing | Amicus Therapeutics
SEC Filing | Amicus Therapeutics

Amicus Therapeutics Provides Updates for AT-GAA for Pompe Disease -  Canadian Association of Pompe
Amicus Therapeutics Provides Updates for AT-GAA for Pompe Disease - Canadian Association of Pompe

SEC Filing | Amicus Therapeutics
SEC Filing | Amicus Therapeutics

Amicus: An Inflection Point In Growth (NASDAQ:FOLD) | Seeking Alpha
Amicus: An Inflection Point In Growth (NASDAQ:FOLD) | Seeking Alpha

After FDA Discussions, Amicus Therapeutics to Push Pompe Disease Program  into Phase II | BioSpace
After FDA Discussions, Amicus Therapeutics to Push Pompe Disease Program into Phase II | BioSpace

Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease  - ScienceDirect
Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease - ScienceDirect

MDA2021 — Miglustat's Role in AT-GAA, Possible Pompe Therapy, Described
MDA2021 — Miglustat's Role in AT-GAA, Possible Pompe Therapy, Described

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of  Secretable Acid α-Glucosidase - ScienceDirect
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase - ScienceDirect

Enzymatic diagnosis of Pompe disease: lessons from 28 years of experience |  European Journal of Human Genetics
Enzymatic diagnosis of Pompe disease: lessons from 28 years of experience | European Journal of Human Genetics

Pathophysiology of late-onset Pompe disease. Abbreviations: GAA, acid... |  Download Scientific Diagram
Pathophysiology of late-onset Pompe disease. Abbreviations: GAA, acid... | Download Scientific Diagram

Biomolecules | Free Full-Text | Pompe Disease: New Developments in an Old  Lysosomal Storage Disorder
Biomolecules | Free Full-Text | Pompe Disease: New Developments in an Old Lysosomal Storage Disorder

Amicus Therapeutics Provides Updates for AT-GAA for Pompe Disease -  Canadian Association of Pompe
Amicus Therapeutics Provides Updates for AT-GAA for Pompe Disease - Canadian Association of Pompe

FDA Expected to Act Soon on AT-GAA for Pompe Disease - Rare Disease Advisor
FDA Expected to Act Soon on AT-GAA for Pompe Disease - Rare Disease Advisor

GAA activities in the Pompe diseasemouse. Pompe disease mice received a...  | Download Scientific Diagram
GAA activities in the Pompe diseasemouse. Pompe disease mice received a... | Download Scientific Diagram

Correction of oxidative stress enhances enzyme replacement therapy in Pompe  disease | EMBO Molecular Medicine
Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease | EMBO Molecular Medicine

Pompe disease: pathogenesis, molecular genetics and diagnosis | Aging
Pompe disease: pathogenesis, molecular genetics and diagnosis | Aging

Amicus Therapeutics Announces Positive Long-Term Data from
Amicus Therapeutics Announces Positive Long-Term Data from

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in  a novel mouse model with early-onset spinal cord and respiratory defects -  eBioMedicine
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects - eBioMedicine

FDA Extends Review Of Amicus Therapeutics' AT-GAA For The Treatment Of Pompe  Disease By Three Months | Markets Insider
FDA Extends Review Of Amicus Therapeutics' AT-GAA For The Treatment Of Pompe Disease By Three Months | Markets Insider

Molecules | Free Full-Text | Pharmacological Chaperone Therapy for Pompe  Disease
Molecules | Free Full-Text | Pharmacological Chaperone Therapy for Pompe Disease

Pombiliti + Opfolda for late-onset Pompe disease | Pompe Disease News
Pombiliti + Opfolda for late-onset Pompe disease | Pompe Disease News

Correction of GAA activity in Pompe disease fibroblasts. (A) To study... |  Download Scientific Diagram
Correction of GAA activity in Pompe disease fibroblasts. (A) To study... | Download Scientific Diagram