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.

Scientific Advisory Board

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.

メーリングリストに参加する

Scientific Advisory Board

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.

メーリングリストに参加する

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.