Related Experiment Videos
Corrigendum to "Pompe disease: Unmet needs and emerging therapies" [Mol Genet Metab. 2024 Nov;143(3):108590]
Kelly A George1, Allyson L Anding1, Arjan van der Flier1
1Sanofi, Cambridge, MA, USA.
Molecular Genetics and Metabolism
|March 11, 2025
Abstract
No abstract available in PubMed .
Related Concept Videos
Articles linked to this work by shared authors, journal, and citation graph.
Accelerating Medicines Partnership® Parkinson's Disease Proteomics: A Comprehensive Resource for Advancing Parkinson's Disease Research.
Movement disorders : official journal of the Movement Disorder Society·2026
AAV gene therapy for GBA1-related diseases.
Molecular therapy : the journal of the American Society of Gene Therapy·2025
Pharmacodynamics, Safety, and Tolerability of Pyridostigmine Bromide in Heart Failure.
Current therapeutic research, clinical and experimental·2025
Protein Assembly Modulation: A New Approach to Amyotrophic Lateral Sclerosis (ALS) Therapeutics.
Journal of experimental neurology·2025
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1.
Molecular therapy : the journal of the American Society of Gene Therapy·2025
Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice.
Molecular therapy. Methods & clinical development·2025
Metabolic alterations associated with immune dysfunction in lysinuric protein intolerance.
Molecular genetics and metabolism·2026
Population-based newborn screening for inherited metabolic diseases in Beijing, China: findings from 404,990 infants.
Molecular genetics and metabolism·2026
Longitudinal biochemical profiles in beta-ketothiolase deficiency: Phase-dependent diagnostic challenges and metabolic variability.
Molecular genetics and metabolism·2026
Bone mineral density in participants with phenylalanine hydroxylase (PAH) deficiency: a report from the PHEFREE rare disorders consortium.
Molecular genetics and metabolism·2026
Human cell-based models for urea cycle research: From gene regulation and ureagenesis to disease modeling and therapeutic development.
Molecular genetics and metabolism·2026
[Development of universal off-the-shelf T cell therapies derived from ES/iPS cells for leukemia and COVID-19].
[Rinsho ketsueki] The Japanese journal of clinical hematology·2026
[Intrathecal immunoglobulin administration for refractory viral central nervous system infection after allogeneic hematopoietic stem cell transplantation].
[Rinsho ketsueki] The Japanese journal of clinical hematology·2026
Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis With Cardiomyopathy: Insights From HELIOS-B.
Journal of the American College of Cardiology·2026
Advanced therapy medicinal products in Saudi Arabia: Regulation and development.
Regenerative therapy·2026