Shaghig leads the Clinical and Medical QA and Compliance function for Shire. She has 25 years experience in quality systems, regulatory compliance, HIS and operational mngt. Prior to joining Shire, she held senior managment position at Halloran Consulting and VP, WW Technology Implementation at PAREXEL. She holds a B.S. in HIS from Northeastern and an MBA from Regis Univerity.

Steve leads the development and execution of the TS Alliance's scientific strategy through partnerships and conversations with stakeholders including individuals and families affected by TSC, basic and clinical researchers, healthcare providers, government agencies, and other non-profit organizations. As CSO since 2011, he has led the creation and growth of the TSC Preclinical Consortium to accelerate testing of potential new treatments and the Biosample Repository to share human TSC biosamples. Prior to joining the TS Alliance, Steve was a project leader responsible for driving global project teams toward new human proof-of-concept studies to re-purpose clinical-stage Pfizer compounds for new indications.

David Schubert is the Vice President of Regulatory and Quality at Stealth BioTherapeutics Inc. Previously, Mr. Schubert held positions at MediVector Inc., Logical Therapeutics Inc., Coley Pharmaceutical Group, Genzyme and Cubist Pharmaceuticals. Mr. Schubert has over 30 years of experience in biotechnology regulatory, rare disease drug development, quality, process development, and research and development and has chaired several Oligo-based Therapeutics Conferences co-sponsored by DIA and FDA.

Scott Schliebner is a clinical drug development executive with a 25+ year background, specializing in rare diseases and orphan drug development. His experience encompasses all trial phases; a broad variety of study designs; interventional and observational studies; across a diverse range of rare indications. Mr. Schliebner has held leadership positions within the biotech, non-profit, and CRO sectors, and currently serves on the Board of Uplifting Athletes, where he also leads the Upifting Athletes Scientific Advisory Board. Additionally, Mr. Schliebner serves as Co-Chair of Global Genes' Corporate Alliance and is committed to patient-focused approaches to accelerate the development of new rare disease therapies.

Linda currently serves as PCOA Lead working across Pfizer’s therapeutic categories addressing efforts to develop, validate and use COAs as endpoints for providing evidence of medical treatment benefit differentiation, labeling and value. Linda has served patients and the pharmaceutical industry for the past 20 years in similar roles at Wyeth, Janssen, Shire and Pfizer. Linda has developed and/or validated COAs across multiple therapeutic categories. Linda is trained as a statistician.

Dr. Beetsch, VP of Patient Advocacy in Celgene’s Corporate Affairs Dept., leads the global development & execution of a coordinated patient-focused Advocacy strategy working w/multiple patient, provider, payer, & policy organizations around the world to foster safe & effective solutions to healthcare challenges.

Jessica is a senior regulatory professional and consultant with 10 years of experience in the global biopharmaceutical industry. She provides strategic product development and approval strategies to pharmaceutical and biotechnology companies with a focus on investigational therapies for the treatment of rare, serious and life-threatening conditions.

Larry Bauer is an independent rare disease drug development consultant. Prior to that, he was an FDA Regulatory Scientist in the Center for Drug Evaluation and Research, Office of New Drugs Immediate Office (CDER, OND IO) in the Rare Diseases Program. He was one of the original members of the Rare Diseases Program where he worked from 2010-2018. In that role, he provided regulatory expertise internally and externally regarding FDA laws, regulations, guidances and policies and supports the overall work of the Rare Diseases Program. Prior to coming to the FDA, he worked for 17 years at the National Institutes of Health Clinical Center.

Dr. Papadopoulos serves as the Deputy Director (acting) of the Division of Clinical Outcome Assessment in the Office of New Drugs in the Center for Drug Evaluation and Research (CDER). The Division provides consultation to CDER’s Review Divisions as well as other FDA Centers on clinical outcome assessments (COAs) regarding their development, validation, interpretation and overall suitability to support labeling claims and also manages the COA drug development qualification program.

Dr. Larry Blankstein is an independent Senior Consultant to the Pharmaceutical and Biotechnology industry. He has over 22 years' experience in drug development strategy, clinical operations and mergers and acquisitions. He received his PhD degree in Biology from Wesleyan University in Middletown, CT. His industry drug development experience was at Quintiles, Seragen, and Genzyme.

Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his medical degree from NYU School of Medicine, received post-graduate training in Internal Medicine at Vanderbilt University Hospitals, and completed specialty training in hematology at the University of North Carolina.