Peter B. Kang MD, FAAN, FAAP

 

Dr. Kang is a pediatric neurologist and physician-scientist whose clinical and research interests focus on neurogenetic disorders, including Cockayne syndrome.  He is Director of the Paul & Sheila Wellstone Muscular Dystrophy Center and Vice Chair of Research for the Department of Neurology at the University of Minnesota Medical School.

Dr. Kang’s research laboratory focuses on the genetics of limb girdle muscular dystrophy and other pediatric neuromuscular disorders, along with the mechanisms of rare muscle diseases including MEGF10 myopathy, and he has published extensively on these subjects.  His work has been supported by the National Institutes of Health (NIH), the Centers for Disease Control and Prevention (CDC), the Muscular Dystrophy Association, and the Xtraordinary Joy Foundation.  Among his former trainees, several are faculty members in the United States, Canada, South Korea, and Japan.

Dr. Kang is an associate editor of Muscle & Nerve and an editorial board member of two other journals: Neurology and Pediatric Neurology.  He served two terms on the editorial board of Pediatrics.  Prior to his arrival at the University of Minnesota in 2021, Dr. Kang was Chief of the Division of Pediatric Neurology and Professor of Pediatrics at the University of Florida College of Medicine.  Earlier in his career, he was the Director of the Electromyography Laboratory at Boston Children’s Hospital and Associate Professor of Neurology at Harvard Medical School.

 

Amy L. Marini RN, BSN, CHTP


Amy has been a registered nurse focused on pediatric nursing for over 18 years.  Her passion to help children has been lifelong.  Her clinical experience started with staff nursing then transitioned to the role of clinical education specialist at the largest pediatric rehabilitation facility in the country for 13 years.  This coupled with ongoing private duty in home pediatric nursing has prepared her for the most important nursing role yet:  mom of three, two of whom have Cockayne Syndrome.  Since the diagnosis of her children in 2015 she has focused on creating awareness for Cockayne Syndrome and strives to help other families understand the complexities of the diagnosis so they can in turn advocate and help their children as much as possible.

Amy has her Bachelors in Nursing from William Paterson University and is also a Certified Healing Touch Practitioner.

 

Andrew E. Mulberg MD, FAAP, CPI

 

Andrew is currently an Executive pharmaceutical leader leading strategy and operational aspects of drug and biological development for NDA and BLA submissions for pediatric and adult rare diseases, cystic fibrosis, gastroenterology and hepatology. He is currently Senior Vice President, Regulatory Affairs at Neurogene Inc, a company devoted to gene therapy approaches to management of neurodegenerative disorders in children and adults.

Formerly, he served Senior Vice President, Global Regulatory Affairs at Amicus Therapeutics from 2016-2020 and responsible for the approval of Galafold (migalastat) for the treatment of Fabry disease in adults. He has been involved in the registration planning for AT-GAA, a novel enzyme replacement therapy for Pompe disease in infants, children and adults.  He served as Division Deputy Director of Gastroenterology and Inborn Errors Products, Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA) since 2010-2016. 

Before joining FDA, Andrew was Portfolio Leader in Established Products responsible for worldwide leadership of Internal Medicine products in Johnson and Johnson from 2000-2010. 

Andrew is a graduate of Columbia College of Columbia University and of the Mount Sinai School of Medicine.  He completed his residency in Pediatrics at the Children’s Hospital of Philadelphia followed by a Pediatric Gastroenterology Clinical Fellowship and a Post-Doctoral Fellowship in Cellular and Molecular Physiology at New England Medical Center.  Andrew is Adjunct Professor of Pediatrics at the University of Maryland School of Medicine and has served as Attending, Pediatric Gastroenterology and Nutrition at Cooper University Hospital in New Jersey caring for children with gastrointestinal diseases.  He is Principal Editor of Pediatric Drug Development: Concepts and Applicationspublished in 2011 and 2013.  He is a member of Alpha Omega Alpha Honor Medical Society, American Gastroenterological Association and the North American Society for Pediatric Gastroenterology and Nutrition.

 

Christina A. Pacak PhD

 

Christina A. Pacak, PhD. is a translational molecular biologist with a long-standing personal interest in Cockayne syndrome. She has built a laboratory program to develop gene therapies for several forms of Cockayne syndrome and to gain a greater understanding of the underlying disease mechanisms.

Dr. Pacak is an Assistant Professor of Neurology at the University of Minnesota Medical School, and was previously an Assistant Professor of Pediatrics at the University of Florida College of Medicine. She has served on national committees for the American Society of Gene and Cell Therapy and has published over a dozen peer-reviewed manuscripts describing the development or use of adeno-associated virus (AAV) as a gene delivery system to treat rare diseases.

Dr. Pacak earned her PhD at the University of Florida and completed a postdoctoral fellowship at Boston Children’s Hospital – Harvard Medical School.

 

Kristin Dayton MD

 

Dr. Kristin Dayton earned her medical degree from Wake Forest University School of Medicine and completed her residency in general pediatrics at the University of Florida in Gainesville, Florida. She completed her fellowship training in pediatric endocrinology at the University of Florida where she now serves as an Assistant Professor in the Department of Pediatric Endocrinology. She is a Diplomate of the American Board of Pediatrics with board certification in Pediatrics and Pediatric Endocrinology.

Dr. Dayton is a member of the Pediatric Endocrine Society.

 

 

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