Dr. Pearce heads the leading lab in Juvenile Batten disease research. He has been researching Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease) since 1997. His research has led to the first clinical trial for Juvenile Batten disease. He has published over 100 research papers on Batten disease. He also oversees a national registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS). He has served on numerous NIH review committees, has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH) and is currently a member of the executive committee for the International Rare Diseases Research Consortium (IRDiRC).

Dr. Ramirez trained as a pediatrician in her native Costa Rica and moved to Rochester, NY in 2000 to pursue a Master in Public Health followed by a PhD in Genetics. She later completed residency training in Child Neurology in 2012. She is one of the small number of clinician scientists who has been involved in basic science and clinical research in the field of Batten Disease. ​ Upon finishing her residency training, she moved to the University of Virginia, where she worked under the mentorship of known epileptologists Jaideep Kapur MBBS, PhD and Howard Goodkin MD, PhD. She recently moved to The University of Tennessee Medical Center, where she continues to care for children with Batten disease and other neurological conditions.

Russell Gotschall brings over 25 years of drug discovery and development to M6P Therapeutics. Prior to joining M6P, Russell was VP of R&D at Amicus Therapeutics, where he managed pre-clinical R&D and helped build a world-class discovery and research organization that maximizing protein engineering and analytics capabilities in the developing of a robust pipeline of novel gene therapy programs in collaboration with the University of Pennsylvania. Russell has had a long career in the clinical and biopharmaceutical industry utilizing his glycobiology expertise to support the development of analytical assays to understand pediatric pharmacology and to support drug development of advanced therapeutics for rare diseases. His experience spans multiple indications including lysosomal storage diseases, cardiovascular, mineral metabolism disorders and chronic kidney disease. Russell is a graduate of Wichita State University with a Master Degree in Biology with an emphasis in glycobiology.

Gavin has more than 30 years of biopharmaceutical industry experience with deep experience in developing therapeutics for rare diseases. Most recently he led the development of EXONDYS 51 for the treatment of Duchenne muscular dystrophy (DMD). His rare disease expertise and patient centric focus came from twenty-years with Genzyme, where he led the global development of therapeutics for Mucopolysaccharidosis type I (MPS I), Pompe and Gaucher

Dr. Kruer is a pediatric neurologist and developmental pediatrician at Phoenix Children’s Hospital, where he serves as director of the Cerebral Palsy & Pediatric Movement Disorders Program. He is associate professor of Child Health, Cellular & Molecular Medicine, Genetics, and Neurology at the University of Arizona College of Medicine Phoenix. Michael’s clinical and research work focuses on improving the health and quality of life of children with neurodevelopmental and neurodegenerative disorders. Dr. Kruer is a physician-scientist, treating children with neurologic and genetic disorders in the hospital and clinic while working to understand the neurobiological basis of disease in the lab. Dr. Kruer’s research team is dedicated to understanding how genetic mutations lead to cerebral palsy, movement disorders, and rare diseases. His talented lab members include computational scientists, molecular and cellular biologists, neuroscientists, physicians, and clinical researchers working toward the common goal of deciphering the molecular mechanisms that lead to childhood neurological disease in order to improve both diagnosis and treatment. Michael’s research has been supported by the American Academy of Cerebral Palsy & Developmental Medicine, Dystonia Medical Research Foundation, Child Neurology Foundation, and the US National Institutes of Health, among others. He is a past recipient of a Clinical Scientist Development Award from the Doris Duke Foundation and the Shields Award from the Child Neurology Foundation. Dr. Kruer is chair of the International Cerebral Palsy Genomics Consortium, an international collaborative effort that uses cutting-edge genomic findings as a window into the causes of cerebral palsy. He is delighted to be a part of the scientific advisory board of ForeBatten.