Ozlem Goker-Alpan, MD
Founder and CMO of LDRTC
Ozlem Goker-Alpan, MD, has over 20 years of experience in the rare diseases field. She is a world-renowned clinician and translational scientist in rare genetic and lysosomal storage disorders, training a new generation of physicians and advocacy patient groups. At LDRTC, Dr. Goker-Alpan oversees multiple scientific projects exploring immune pathways and lysosomal functions to develop new diagnostic and monitoring tools in LSDs and GBA-related Parkinsonism.
Email: ogoker-alpan@ldrtc.org
My PublicationsEmail: ogoker-alpan@ldrtc.org
My Work
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Recent Publications •
1. Goker-Alpan O, Ivanova MM, Pathak R, Wright. Enzyme replacement therapy in infants and very young children with Gaucher disease using velaglucerase alfa: a single-center experience. E.Front Pediatr. 2025 Oct 17;13:1613599. doi: 10.3389/fped.2025.1613599. eCollection 2025.
2. Ivanova MM, Dao J, Kasaci N, Huang F, Nguyen E, Goker-Alpan O. Age-related inflammatory biomarkers in early-onset osteoporosis in females with Gaucher disease. Front Endocrinol (Lausanne). 2025 Jul 2;16:1606218. doi: 10.3389/fendo.2025.1606218. eCollection 2025.
3. Ivanova MM, Dao J, Friedman A, Kasaci N, Goker-Alpan O. Sex Differences in Circulating Inflammatory, Immune, and Tissue Growth Markers Associated with Fabry Disease-Related Cardiomyopathy. Cells. 2025 Feb 20;14(5). PubMed PMID: 40072051.
4. Svarny L, Agha A, Dao J, Sandhu B, Ivanova M, Goker-Alpan O. The clinical utility of neurofilament light chain for early detection and prediction of disease burden and severity in neuronopathic Gaucher disease. Mol Genet Metab. 2025 Aug;145(4):109181. doi: 10.1016/j.ymgme.2025.109181. Epub 2025 Jun 19. PMID: 40570595.
5. Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. J Med Genet. 2023 Nov 8:jmg-2023-109445. doi: 10.1136/jmg-2023-109445.
