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Scientific Advisory Board
W H O ' S I N O U R C O R N E R
The scientists behind the search for a cure.
The Scientific Advisory Board for NLSDM was established to offer expertise on scientific developments, to provide research oversight and assure that Gabby's Wonderful World policies and communications meet the highest standards of scientific rigor and accuracy.
Paul T. Martin, PhD
Jerry R. Mendell Center for Gene Therapy, Nationwide Children's Hospital; Professor, Ohio State
Leading Principal Investigator developing a gene therapy for NLSDM.
Kajus Merkevičius, PhD
Biomedical researcher; trained with the Morava-Kozicz group at Mount Sinai
Rare metabolic and mitochondrial disorders. Participating in the NLSDM drug screening project.
Michio Hirano, MD
Lucy G. Moses Professor of Neurology and Chief of Neuromuscular Medicine, Columbia University
Internationally recognized in mitochondrial disease, genetic myopathies and new therapies.
Hasan Orhan Akman, PhD
Columbia University
Diagnosis and treatment of metabolic disorders that cause neuromuscular disease.
Eva Morava, MD, PhD
Professor of Genetics and Genomic Sciences and Pediatrics, Icahn School of Medicine at Mount Sinai
Leading researcher in rare metabolic disorders. Conducting a drug screening project for NLSDM.
Ken-ichi Hirano, MD, PhD
Specially Appointed Professor, Department of Triglyceride Science, The University of Osaka
Discovered the triglyceride disease TGCV and leads development of the investigational drug CNT-01.
Dr. Einat Zalckvar
Principal Investigator, Zalckvar Lab, Bar-Ilan University, Israel
Studies how defects in lysosomal function and lipid handling contribute to rare metabolic disease.
Gustavo H. B. Maegawa, MD, PhD, FACMG
Columbia University
Leads the Maegawa Lab, focused on lysosomal storage diseases.
Simona Treidler, MD
Stony Brook Medicine
Physician specializing in neuromuscular medicine.
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