Patrick B. Deegan
Cambridge, ENG, United Kingdom
Last Updated: 09/26/2026
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Found 62 publications
A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.
Journal: Journal of comparative effectiveness research
Published: July 15, 2026
Repurposing Gaucher disease therapy for Saposin C deficiency: Proof-of-concept with eliglustat.
Journal: Molecular genetics and metabolism
Published: December 16, 2025
Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study.
Journal: Orphanet journal of rare diseases
Published: November 12, 2025
Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry.
Journal: Journal of inherited metabolic disease
Published: October 09, 2025
Multimodal phenotypic clustering predicts cardiac outcomes in Fabry disease.
Journal: Orphanet journal of rare diseases
Published: August 15, 2025
Diagnosis, management and monitoring of patients with Pompe disease in the UK.
Journal: BMJ neurology open
Published: July 29, 2025
Historical Control Analysis Demonstrates Greater Long-Term Reduction in Plasma Globotriaosylceramide (Gb3) by Venglustat Compared With Placebo or Agalsidase Beta in Male Patients With Classic Fabry Disease.
Journal: Molecular genetics & genomic medicine
Published: April 14, 2025
Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry.
Journal: Journal of clinical medicine
Published: April 19, 2024
Clinical management of female patients with Fabry disease based on expert consensus.
Journal: Orphanet journal of rare diseases
Published: March 28, 2024
Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.
Journal: Journal of clinical medicine
Published: March 25, 2024
A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies.
Journal: Journal of inherited metabolic disease
Published: February 23, 2024
Last Updated: 09/26/2026