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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.

Email: ogoker-alpan@ldrtc.org
My Publications

My Work

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Recent Publications •

  1. Ivanova MM, Dao J, Svarny L, Nguyen E, Ponnam S, Goker-Alpan O. NGAL as a Biomarker of Tubular Injury and Glomerular Dysfunction in Fabry Nephropathy. Cells. 2026 Oct 1;15(19):1797. doi: 10.3390/cells15191797. PMID: 42882495.
  2. di Ronza A, Bratkovic D, Byrne BJ, Clemens PR, Goker-Alpan O, Kishnani PS, Mozaffar T, Roberts M, Van Der Beek NA, Amon F, Fox B, Holdbrook F, Jain V, Schoser B. 90-month Muscle Function and Biomarker Outcomes With Cipaglucosidase Alfa Plus Miglustat (Cipa+Mig) in Adults With Pompe Disease in ATB200-02, an Open-label Phase I/II Study (S7.006). Neurology. 2026 Jun 9;106(11 Suppl 1):2056. doi: 10.1212/WNL.0000000000215573.
  3. Hiwot T, Porter FD, Bremova-Ertl T, Ramaswami U, Hastings C, Héron B, Hopkin J, Melville J, Amartino H, Del Toro M, Deodato F, Ezgü F, Gissen P, Gibson JB, Ficicioglu C, Giugliani R, Staretz-Chacham O, Platt F, Guffon N, Julich K, Kresojević N, Lehman A, Nadjar Y, Schneider SA, Jones S, Mengel E, Tchan M, Walterfang M, Goker-Alpan O, Dawson C, Cowie S, Mathieson T, Berry-Kravis E, Patterson MC. 2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C. J Inherit Metab Dis. 2026 May;49(3):e70185. doi: 10.1002/jimd.70185. PMID: 42036217.
  4. Holida M, Linhart A, Longo N, Wallace E, Tøndel C, Hughes D, Warnock DG, Pisani A, Eyskens F, Deegan P, Feldt-Rasmussen U, Goker-Alpan O, Mehta A, Piotti G, Fichera V, Wang M, Chertkoff R, Waldek S, Wilcox WR, Bernat JA. 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. Orphanet J Rare Dis. 2026 Mar 20;21(1):185. doi: 10.1186/s13023-026-04303-8. PMID: 41862950.
  5. Revel-Vilk S, Deegan P, Day-Salvatore D, Goker-Alpan O, Mengel E, Batista JL, Carwile JL, Perichon G, Ficicioglu C. 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. J Inherit Metab Dis. 2026 Mar;49(2):e70161. doi: 10.1002/jimd.70161. PMID: 41714274.