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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.

My Publications

My Work


Recent Publications •

1. 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.

2. Banerjee D, Ivanova MM, Celik N, Kim MH, Derman ID, Limgala RP, Ozbolat IT, Goker-Alpan O. Biofabrication of an in-vitro bone model for Gaucher disease. Biofabrication. 2023 Sep 22;15(4):045023. doi: 10.1088/1758-5090/acf95a. PMID: 37703870; PMCID: PMC10515412.

3. Weinreb NJ, Goker-Alpan O. Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent. JAMA Netw Open. 2023 Jun 1;6(6):e2319336. doi: 10.1001/jamanetworkopen.2023.19336. PMID: 37342045.

4. Ivanova MM, Dao J, Slayeh OA, Friedman A, Goker-Alpan O. Circulated TGF-β1 and VEGF-A as Biomarkers for Fabry Disease-Associated Cardiomyopathy. Cells. 2023 Aug 19;12(16):2102. doi: 10.3390/cells12162102. PMID: 37626912; PMCID: PMC10453505.

5. Hughes DA, Deegan P, Giraldo P, Göker-Alpan Ö, Lau H, Lukina E, Revel-Vilk S, Scarpa M, Botha J, Gadir N, Zimran A; GOS Steering Committee. Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158". J Clin Med. 2023 Jun 13;12(12):4017. doi: 10.3390/jcm12124017. PMID: 37373710; PMCID: PMC10299381.