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Ozlem Goker-Alpan (born in 1966) is the Founder and Chief Medical Officer of the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC). She founded the LDRTC in 2013 to provide high-quality care for individuals with lysosomal storage diseases and other rare genetic disorders.

Early Life
Ozlem Goker-Alpan was born and raised in Istanbul, Turkey.

Education
Ozlem Goker-Alpan completed her medical degree at Marmara University School of Medicine in 1990. She pursued her residency in Pediatrics at the same institution from 1990 to 1992 and continued her residency at SUNY at Stony Brook in New York from 1992 to 1996. Goker-Alpan further specialized by completing a fellowship in Clinical and Biochemical Genetics at the Greater Washington Medical Genetics Fellowship Training Program, NIH, Bethesda, MD, from 1996 to 1999. She completed a second fellowship focused on Lysosomal Storage Disorders and Gaucher Disease at the Clinical Neuroscience Branch NIMH, after which she coordinated the NIH Gaucher Clinic at the Medical Genetics Branch of the National Human Genome Research Institute.

Career and Research
Dr. Goker-Alpan is the Chief Medical Officer of the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), which she founded in 2013. Her primary research focus is on improving treatments for individuals with rare genetic disorders, particularly lysosomal storage diseases.

Gaucher Disease: Dr. Goker-Alpan's research in Gaucher Disease encompasses clinical trials assessing new therapies' efficacy and safety, identifying biomarkers for better patient management, and exploring disease mechanisms.

Pompe Disease: Her work also includes significant contributions to Pompe Disease research, investigating long-term safety and efficacy of therapies like Avalglucosidase Alfa and evaluating patient outcomes during pregnancy.

Fabry Disease: In Fabry Disease, Dr. Goker-Alpan's studies focus on biomarkers for disease-associated cardiomyopathy and the safety and efficacy of treatments, including oral chaperone therapy.

The research on Gaucher disease focuses on several critical areas to enhance understanding and treatment of the condition. These areas include conducting clinical trials to assess the efficacy and safety of emerging therapies for Gaucher disease, investigating potential biomarkers to improve patient diagnosis, monitoring, and personalized treatment strategies, and exploring the underlying mechanisms of Gaucher disease to uncover new therapeutic targets and understand disease progression. By addressing these aspects, The research has contributed to better clinical outcomes and quality of life for patients with Gaucher disease.

Patents
2022: Patient-specific bone model in Gaucher disease using 3D Bioprinting.

2021: Inhibition of caspase pathway for Lysosomal storage disorders.

2020: Ambroxol to treat Sanfilippo disease.

2017: Protein markers for diagnosis of GBA-associated Parkinson's disease.

Positions and Appointments
2013-Present: Founder and Chief of Medical Lysosomal & Rare Disorders Research & Treatment Center, Fairfax, VA

2010-2017: Director, Lysosomal Disorders Unit and Center for Clinical Trials, O&O Alpan LLC, Fairfax, VA

2010-2012: Special Consultant, NHGRI, NIH, Bethesda, MD

2005-2009: Senior Staff Clinician, MGB/NHGRI, NIH, Bethesda, MD

2001-2005: Senior Staff Fellow on Lysosomal Storage Disorders and Gaucher Disease, Section on Molecular Neurogenetics, NIMH, NIH, Bethesda, MD

1998-1999: Adjunct Scientist, Hereditary Disorders Branch, NICHD, NIH, Bethesda, MD

1996-1998: Clinical Fellow, Medical Genetics Branch, NHGRI, NIH, Bethesda, MD

Professional Memberships

 * European Society of Gene and Cell Therapy
 * American College of Medical Genetics
 * Society for Neuroscience
 * Society for the Study of Inborn Errors of Metabolism
 * Society of Pediatric Research
 * Society of Inherited Metabolic Diseases
 * Turkish Society of Hematology (Honorary Member)
 * Movement Disorder Society
 * American Society of Human Genetics

Selected Publications
''Weinreb NJ, Goker-Alpan O. (2023). "Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent." JAMA Netw Open.''

''Weinreb NJ, Goker-Alpan O, et al. (2022). "The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?" Mol Genet Metab.''

''Ivanova MM, Dao J, Kasaci N, et al. (2021). "Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease." PLoS One.''

''Schiffmann R, Sevigny J, Rolfs A, et al. (2020). "The definition of neuronopathic Gaucher disease." J Inherit Metab Dis.''