OCTOBER 16-17, 2017 MARRIOTT WARDMAN PARK

DAY 1MONDAY, OCTOBER 16, 20177:00 Conference Registration and Continental Breakfast8:00 NORD’S WELCOME & SUMMIT PREVIEWHeather Golding, Vice President Legal and Compliance,Sobi North AmericaGina Parziale, C.F.R.E., Executive Director,Alport Syndrome FoundationMathieu Boudes, Operations & Projects Manager, EURORDISPeter L. Saltonstall, President and Chief Executive Officer, NORDMarshall L. Summar, M.D., Director, Children’s National RareDisease Institute and Chief, Genetics and Metabolism,Children’s National Medical Center; Chairman, Board10:15 Networking & Refreshment Breakof Directors, NORDNETWORKING APPOINTMENT TIMES AVAILABLE8:15 COMMUNITY KEYNOTE ADDRESSMike Porath, Founder and CEO, TheMighty.com9:00 FDA KEYNOTE ADDRESSScott Gottlieb, M.D., Commissioner, FDA9:30 ETHICAL GUIDELINES FOR PATIENT ORGANIZATIONS &INDUSTRY TO COLLABORATEHow can patient organizations and pharmaceutical companies work together toward shared goals withoutcompromising ethical standards?Moderator: Arthur Caplan, M.D., Ph.D., Drs. William F. andVirginia Connolly Mitty Chair Director, Division of MedicalEthics, NYU Langone Medical CenterFireside Chat:Henry R. Moehring, President and CEO, Alpha-1 Foundation11:00 ASSURING PATIENT ACCESS: FUTURE OUTLOOK FORPATIENT ASSISTANCE PROGRAMSAs Patient Assistance Programs come under fire, what canbe done to assure patient access to lifesaving treatmentsfor rare diseases?Moderator: Jayson Slotnik, J.D., Partner, Health PolicyStrategies, Inc.Panelists:Erin Tite, Parent of patientAnne Willis, M.A. Senior Director, Policy and Advocacy,Cystic Fibrosis FoundationBill Schultz, Partner, Zuckerman Spaeder, LLPRob Metz, Senior Vice President, Business Operations &External Affairs, Horizon Pharma12:15 LUNCH & LEARN BREAKOUT ROUNDTABLESSponsor booths open and poster presenters available at their posters; Reserve your seat when registering.1RARE CANCER ORGANIZATION NETWORKINGJim Palma, Executive Director, TargetCancerFoundation2THE PROMISE OF IMMUNOTHERAPY FORCANCER PATIENTS3ATTRACTING YOUNG RESEARCHERS TORARE DISEASESAnne Pariser, M.D., Deputy Director, Office ofRare Diseases Research, NCATS, NIH4WHAT’S AHEAD FOR PERSONALIZED MEDICINE?Philip John (P.J.) Brooks, Ph.D., Program Director,Division of Clinical Innovation, NCATS, NIH9 DEVICES FOR RARE PEDIATRIC DISEASESVasum Peiris, M.D., M.P.H., Chief Medical Officerof Pediatrics and Special Populations, Center forDevices and Radiological Health, FDA10SAFETY ISSUES IN RARE DISEASEDRUG DEVELOPMENTKathryn O’Connell, M.D., Ph.D., Medical Officer,Rare Diseases Program, Office of New Drugs,CDER, FDA11RARE PEDIATRIC DISEASE PRIORITY REVIEWVOUCHERS & DESIGNATIONSLarry Bauer, Regulatory Scientist, Rare DiseasesProgram, Office of New Drugs, CDER, FDAAlthea Cuff, Senior Regulatory Health ProjectManger, Rare Diseases Program, Office of NewDrugs, CDER, FDACarla Epps, M.D., M.P.H., Medical Officer, CDER, FDA5ASSURING PATIENT ACCESS THROUGH PAPsJenica Stroock, Director, CorporateResponsibility, Pfizer6CDER ENGAGEMENT WITH PATIENT12 NAVIGATING CBER ADVOCACY GROUPSDiane Maloney, J.D., Associate Director for Policy,Scott Winiecki, M.D., Team Lead, ProfessionalCBER, FDAAffairs and Stakeholder Engagement, CDER, FDA Julienne Vaillancourt, R.Ph., M.P.H., CAPT, USPHS CC, Policy Advisor and Rare Disease Liaison,INCENTIVES FOR DEVELOPINGOffice of the Center Director, Associate DirectorORPHAN PRODUCTSfor Policy, CBER, FDAGayatri Rao, M.D., J.D., Director, Office ofOrphan Products Development, FDA13 GENE THERAPY POLICIES AT CBER78NATURAL HISTORY STUDIESGumei Liu, M.D., Ph.D., Office of OrphanProducts Development, FDAVanessa Boulanger, Director of Research Programs, NORDAllison Seebald, Research ProgramsAssociate, NORDIlan Irony, M.D., Deputy Director Divisionof C linical Evaluation and Pharmacology/ Toxicology, CBER, FDA1415CREATIVE PROTOCOL DESIGNS TO ACCELERATE REGULATORY APPROVALSJules T. Mitchel, MBA, Ph.D., President,Target Health Inc.ASSESSING THE VALUE OF ORPHAN DRUGSDennis Jackman, Senior VP, Public Affairs, CSLBehring Biopharmaceuticals16A CONVERSATION WITH JONATHAN GOLDSMITH, CDER RARE DISEASE OFFICEJonathan C. Goldsmith, M.D., F.A.C.P., AssociateDirector Rare Diseases Program, Office of NewDrugs, CDER, FDA17BOARD MANAGEMENT FORPATIENT ORGANIZATIONSMeegan Carey, Executive Director, PSC Partners18MANAGING VOLUNTEERS INPATIENT ORGANIZATIONSKristen Angell, Associate Director ofMembership, NORD19SOCIAL MEDIA STRATEGIES TO MAKE YOURMESSAGE HEARDShazia Ahmad, Director, Patient and PhysicianServices, UBC20ROLE OF PATIENT ORGANIZATIONS IN ADVOCATING FOR NEW DRUG APPROVALSTerry Jo Bichell, Foundation for AngelmanSyndrome Therapeutics; A-BOM & AngelmanSyndrome Foundation21EDUCATING MEDICAL PROFESSIONALSABOUT RARE DISEASESAmy R.U.L. Calhoun, M.D., Clinical AssistantProfessor, Division of Medical Genetics, SteadFamily Department of Pediatrics, University ofIowa Carver College of Medicine and MedicalConsultant, Iowa Newborn Screening ProgramRonald J. DeBellis, Pharm.D., FCCP, Chief ScientificOfficer, NORD, Professor of Clinical Sciences,Keck Graduate Institute School of Pharmacy22GENOME SEQUENCING & RAREDISEASE DIAGNOSIS23COMMERCIALIZING GENE THERAPIES5

1:30 CHOOSE ONE OF THREE BREAKOUT SESSIONS (A – C)BREAKOUT ABREAKOUT BPROMOTINGEARLIER DIAGNOSISNEXT-GENERATION TREATMENTS& ADVANCING CLINICAL TRIALSTHE PHYSICIAN PERSPECTIVEDebra Regier, M.D., Ph.D., Director of Education, Children’s National RareDisease InstituteAmy R.U.L. Calhoun, M.D., Clinical AssistantProfessor, Division of Medical Genetics, SteadFamily Department of Pediatrics, Universityof Iowa Carver College of Medicineand Medical Consultant, Iowa Newborn Screening ProgramADVANCING DIAGNOSISMichael Patrick Gray, M.P.H., Senior Director ofMedical Services, Pulmonary HypertensionAssociationDerek Blackway, Senior Manager, Communications & Advocacy, Guthy-JacksonCharitable FoundationADVANCES IN GENETIC TESTINGRobert Nussbaum M.D., Chief Medical Officer,INVITAESUCCESSFUL STRATEGIESFOR PATIENT ORGANIZATIONSTrack Moderator: Philip John (P.J.) Brooks,Ph.D., Program Director, Division of ClinicalInnovation, National Center for AdvancingTranslational Sciences, NIHTrack Moderator: Jennifer Knapp, Executive Director, Adrenal Insufficiency United, O regon Rare Action Network State Ambassador, NORDTHE PROMISE OF GENE THERAPYTHE POWER OF STATE-LEVELLEGISLATIONDavid Lebwohl M.D., Senior VP and GlobalHead for Cell and Gene Therapy, NovartisMaria Kefalas, Co-Founder, CalliopeJoy FoundationSHARE FOR RARE—A COLLABORATIVEPLATFORM MODELPetra Kaufmann, M.D., Director of ClinicalInnovation, National Center for AdvancingTranslational Sciences, NIHDEVELOPMENT OF NOVEL ENDPOINTSUSING MOBILE TECHNOLOGIESTheresa Strong Ph.D., Director of ResearchPrograms, Prader-Willi ResearchLes Jordan, VP, Chief Product Evangelist,Target Health, Inc.2:45 Networking & Refreshment BreakNETWORKING APPOINTMENT TIMES AVAILABLE3:30 THE CHALLENGE OF HEALTHCARE COSTS &TREATMENT PRICESA look at the many factors impacting healthcare costs andtreatment prices today.Moderator: Steve Usdin, Washington Editor, BioCenturyPanelists:Anne McDonald Pritchett, Ph.D., Senior Vice President,Policy & Research, PhRMABill Martin, Vice President, Pharma Strategy and AccountManagement, Express ScriptsJames Geraghty, Entrepreneur in Residence,Third Rock Ventures6BREAKOUT CErica Barnes, Founder/President,Chloe’s Fight Rare Disease Foundation, Minnesota Rare Action Network State Ambassador, NORDJana Monaco, Patient/Family Advisory CouncilChair, Children’s National Medical Center, Children’s National Medical Center, VirginiaRare Action Network State Ambassador,NORDVanessa Puopolo, California Rare Action Network State Ambassador, NORDEFFECTIVE FAMILY PARTNERSHIPSWITH THE MEDICAL CARE TEAMMaryBeth Hollinger, R.N., Director of Education, Support and Advocacy, MitoActionColleen Clarke Muraresku, M.S., C.G.C., Children’s Hospital of Philadelphia4:30 RIGHT TO TRY, CURRENT POLICY NEWS &NORD’S POLICY PRIORITIESModerator: Kate Rawson, Senior Editor, The RPM Report:Regulation, Policy and Market Access and “The Pink Sheet”,Prevision Policy LLCPanelists:Martha Rinker, J.D., VP Policy, NORDKurt R. Karst, Director, Co-Founder and Primary Author ofFDA Law Blog, Hyman, Phelps & McNamara, PCPaul Melmeyer, Director of Federal Policy, NORDTim Boyd, M.P.H., Director of State Policy, NORD5:45 NETWORKING RECEPTION

DAY 2 TUESDAY, OCTOBER 17, 20177:00 Continental Breakfast OpensNETWORKING APPOINTMENT TIMES AVAILABLE7:45 DAY TWO INSIGHTSPeter L. Saltonstall, President and Chief Executive Officer, NORDMarshall L. Summar, M.D., Director, Children’s National RareDisease Institute and Chief, Genetics and Metabolism,Children’s National Medical Center; Chairman, Board ofDirectors, NORD8:00 SUSTAINING ORPHAN DRUG DEVELOPMENTAND AVAILABILITYModerator:Wayne L. Pines, President, Health Care, APCO WorldwidePanelists:Peter L. Saltonstall, President and Chief Executive Officer,NORDMurray Aitken, Senior Vice President and Executive Director,QuintilesIMSMike Lanthier, Operations Research Analyst, Office of theCommissioner, FDAStephen J. Aselage, CEO, Retrophin, Inc.9:00 CHOOSE ONE OF THREE BREAKOUT SESSIONSBREAKOUT SESSION IBREAKOUT SESSION IIBREAKOUT SESSION IIITHE POWER OFDATA-SHARINGDEVELOPING A GLOBAL S TRATEGYFOR RARE DISEASES & ORPHAN DRUGSSUCCESSFUL STRATEGIES FORPATIENT ORGANIZATIONSTrack Moderator: Lewis M. Fredane, M.D.,Clinical Vice President, Bracket GlobalCENTERS OF EXCELLENCEMarshall L. Summar, M.D., Director, Children’s National Rare Disease Instituteand Chief, Genetics and Metabolism, Children’s National Medical Center; Chairman, Board of Directors, NORDPARTICULAR RELEVANCE OF DATA-SHARING TO RARE DISEASESCarrie Wolinetz, Ph.D., Associate Director forScience Policy, NIHSheetal Telang, Senior Director, TherapeuticStrategy, Head of Global Site Identification,Therapeutic Science & Strategy Unit, QuintilesIMSSalvo La Rosa, Vice President Research andDevelopment, Children’s Tumor FoundationTrack Moderator: Durhane Wong-Rieger,CEO, Canadian Organization for Rare Disorders and Chair, Rare Diseases InternationalTrack Moderator: Debbie Drell, Director ofMembership, NORDGLOBAL RESEARCH AND DEVELOPMENT AND ACCESS STRATEGIESValerie Navy-Daniels, Chief Development Officer, Foundation Fighting BlindnessDanielle Pinders, Officer and Event Coordinator, Foundation forAngelman SyndromeKelly Sitkin, Chief Advancement Officer,American Brain Tumor AssociationJulie Raskin, Congential HyperinsulinismInternationalSteve Roberds, Ph.D., Chief Scientific Officer,Tuberous Sclerosis AllianceTHE CURRENT SITUATION IN INDIARamaiah Muthyala, M.D., President & CEO,Indian Organization for Rare DiseasesADDRESSING RARE DISEASES AS AGLOBAL PUBLIC HEALTH CHALLENGEBert Bruce, Vice President Global Marketing,Pfizer Rare DiseaseFUNDRAISING STRATEGIES FOR RAREDISEASE ORGANIZATIONSPCORI FUNDING OPPORTUNITIESAND RESOURCES FOR RARE DISEASE ORGANIZATIONSGyasi Moscou-Jackson, Ph.D., M.H.S., R.N., Program Officer, Science, Healthcare Delivery andDisparities Research, PCORI10:00 Networking & Refreshment BreakJulie Beitz, M.D., Office of Drug Evaluation III, Office ofNew Drugs, CDER, FDANETWORKING APPOINTMENT TIMES AVAILABLE11:40 FDA Perspectives on Gene Therapy10:30 CURRENT TOPICS FROM THE FDAIlan Irony, M.D., D eputy Director Division of ClinicalModerator: Robert Temple, M.D., Deputy Center DirectorEvaluation and Pharmacology/Toxicology, CBER, FDAfor Clinical Science, CDER, FDA12:00 Patient Voice & Engagement with the FDA10:30 FDA/CDER Director Janet Woodcock (via video)Externally-led Patient- Focused Drug DevelopmentJanet Woodcock, M.D., Director, Center for Drug EvaluationMeetings and Lessons Learnedand Research, FDAPujita Vaidya, M.P.H., Office of Strategic Programs, FDA10:40 FDA Leadership Perspectives on Orphan Drug ReviewHow Patients Have Been InvolvedBilly Dunn, M.D., Director, Division of Neurology ProductsAndrea Furia-Helms, M.P.H., Health Programs Coordinator,(DNP), Office of New Drugs, CDER, FDAOffice of Health and Constituent Affairs, FDAWilson Bryan, M.D., Director, Office of Tissues andPatient Engagement in CDRHAdvanced Therapies, CBER, FDAAnindita “Annie” Saha, Director, External Expertise andAmy Barone, M.D., M.S.C.I., Oncology Center of Excellence, FDAPartnerships, CDRH, FDA7

12:30 LUNCH & LEARN BREAKOUT ROUNDTABLESSponsor booths open and poster presenters available at their posters; Reserve your seat when registering.1RARE CANCER ORGANIZATION NETWORKINGJim Palma, Executive Director, TargetCancerFoundation10NAVIGATING EXPANDED ACCESSLucas Kempf, M.D., Medical Officer, Rare DiseasesProgram, Office of New Drugs, CDER, FDA2THE PROMISE OF IMMUNOTHERAPY FORCANCER PATIENTS11RARE PEDIATRIC DISEASE PRIORITY REVIEWVOUCHERS & DESIGNATIONSLarry Bauer, Regulatory Scientist, Rare DiseasesProgram, Office of New Drugs, CDER, FDA317ROLE OF CRITICAL PATH INNOVATION MEETINGS (CPIM) IN RARE DISEASE DRUGDEVELOPMENTChekesh S. Clingman, Ph.D., M.B.A., US PublicHealth Service, Associate Director for StrategicPartnerships, CDER, Office of Translational Sciences, FDAATTRACTING YOUNG RESEARCHERS TORARE DISEASES18 BOARD MANAGEMENT FORAlthea Cuff, Senior Regulatory Health ProjectRashmi Gopal-Srivastava, Ph.D., Director,PATIENT ORGANIZATIONSManager, Rare Diseases Program, office of Extramural Research Program, ORDR, NCATS, NIHMeeganCarey, Executive Director,New Drugs, CDER, FDAPSC Partners4 WHAT’S AHEAD FOR PERSONALIZEDCarla Epps, M.D., M.P.H., Medical Officer, Office of MEDICINE?Orphan Products Development, CDER, FDA19 MANAGING VOLUNTEERS INPhilip John (P.J.) Brooks, Ph.D., Program Director,PATIENT ORGANIZATIONS12 NAVIGATING CBERDivision of Clinical Innovation,, NCATS, NIHKristen Angell, Associate Director of MemberDiane Maloney, J.D., Associate Director for Policy,ship, NORD5 ASSURING PATIENT ACCESS THROUGH PAPsCBER, FDAJenica Stroock, Director, Corporate20 SOCIAL MEDIA STRATEGIES TO MAKE YOURJulienne Vaillancourt, R.Ph., M.P.H., CAPT, USResponsibility, PfizerMESSAGE HEARDPHS CC, Policy Advisor and Rare Disease Liaison,ShaziaAhmad, Director, Patient and PhysicianOffice of the Center Director, Associate Director6 CDER ENGAGEMENT WITH PATIENTServices,UBCfor Policy, CBER, FDA ADVOCACY GROUPSScott Winiecki, M.D., Team Lead, Professional21 ROLE OF PATIENT ORGANIZATIONS IN13 GENE THERAPY POLICIES AT CBERAffairs and Stakeholder Engagement, CDER, FDA ADVOCATING FOR NEW DRUG APPROVALSIlan Irony, M.D., Deputy Director DivisionTerryJo Bichell, Foundation for Angelmanof C linical Evaluation and Pharmacology/ 7 INCENTIVES FOR DEVELOPINGSyndromeTherapeutics; A-BOM & AngelmanToxicology, CBER, FDAORPHAN PRODUCTSSyndrome FoundationGayatri Rao, M.D., J.D., Director, Office of14 CREATIVE PROTOCOL DESIGNS TOOrphan Products Development, FDA22 EDUCATING MEDICAL PROFESSIONALS ACCELERATE REGULATORY APPROVALSABOUT RARE DISEASES8 NATURAL HISTORY STUDIESJules T. Mitchel, MBA, Ph.D., President,AmyR.U.L. Calhoun, M.D., Clinical AssistantGumei Liu, M.D., Ph.D., Office of OrphanTarget Health Inc.Professor,Division of Medical Genetics, SteadProducts Development, FDAFamilyDepartmentof Pediatrics, University of15 ASSESSING THE VALUE OF ORPHAN DRUGSVanessa Boulanger, Director of ResearchIowaCarverCollegeof Medicine and MedicalDennis Jackman, Senior Vice President, PublicPrograms, NORDConsultant,IowaNewbornScreening ProgramAffairs, CSL Behring BiopharmaceuticalsAllison Seebald, Research ProgramsRonald J. DeBellis, Pharm.D., FCCP, Chief ScientifAssociate, NORD16 A CONVERSATION WITH JONATHANic Officer, NORD, Professor of Clinical Sciences, GOLDSMITH, CDER RARE DISEASE OFFICEKeck Graduate Institute School of Pharmacy9 DEVICES FOR RARE PEDIATRIC DISEASESJonathan C. Goldsmith, M.D., F.A.C.P., AssociateVasum Peiris, M.D., M.P.H., Chief Medical OfficerDirector Rare Diseases Program, Office of New23 GENOME SEQUENCING & RAREof Pediatrics and Special Populations, Center forDrugs, CDER, FDADISEASE DIAGNOSISDevices and Radiological Health, FDA242:003:008INVESTOR PERSPECTIVE: THE OUTLOOK FOR INVESTMENT IN ORPHAN PRODUCTSModerator: David Scheer, President, Scheer &Company, Inc.Tony Gibney, Managing Partner, Leerink PartnersKris Jenner, Founding Partner, Rock Springs CaptialJonathan Leff, Partners Private Transactions Team,Deerfield ManagementMaha Katabi, Partner, Sectoral Asset ManagementRARE DISEASE DRUG, BIOLOGIC AND DEVICE DEVELOPMENT: ACHIEVEMENTS AND OPPORTUNITIESModerator: Wayne L. Pines, President, Health Care,APCO WorldwideRichard Moscicki, M.D., Deputy Center Director forScience Operations, CDER, FDACOMMERCIALIZING GENE THERAPIESPeter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, FDAJeffrey Shuren, M.D., J.D., Director, Center for Devices andRadiological Health, FDA4:00 CLOSING REMARKSPeter L. Saltonstall, President and Chief Executive Officer,NORD6:00 MUSIC, MEDICINE AND MOVING FORWARDA Cocktail Reception Hosted by Children’s National RareDisease Institute to celebrate the NORD Summit

POSTERHIGHLIGHTSLife-Transforming TreatmentsAn opportunity throughout each of the networking breaks and luncheons to view original research, innovationsand advancements as numerous posters are displayed, illustrating key themes: INNOVATIVE RESEARCH MEDICAL EDUCATION ADVANCEMENT PATIENT COMMUNITY BUILDING OTHER LIFE-TRANSFORMING TREATMENTS & ADVANCEMENTSRARE TO RARENETWORKINGBringing the Community Closer1:1 Appointments Elevate Networking Opportunities!Maximize your time at the event and build meaningful partnerships by utilizing our appointment-setting softwareto connect with attendees in advance to schedule on-site meetings. Customized profiles for you and your organization Access attendee lists to find potential interesting clients, contacts or partners Software automatically recommends potential matches using keywords Easy scheduling for face-to-face meetings at the event; reserved space handled by NORD Ability to confirm or deny meeting requests Secure, confidential messaging Customize your personal agenda for the conference Automated personal schedule that can be exported to Outlook, iCal, or printed as a PDF Easy access using mobile or desktop9

SPONSORS & EXHIBITORSWho attends?The Summit provides a collaborative environment for researchers from academia, drug and device companies, patient organizations and advocates,policy experts and government organizations responsible for rare disease research and orphan product oversight.BREAKTHROUGH INDUSTRIES & FOCUS AREAS INCLUDE: Patient Services Advocacy Clinical Development R&D Reimbursement Health Policy / Affairs Regulatory Affairs Corporate CommunicationsSponsorship of NORD’s Rare Diseases and Orphan Products Breakthrough Summit exposes your organization to hundreds of stakeholders, d ecisionmakers and influencers committed to the identification, treatment, and the cure of rare diseases. This is through accomplished programs of education, advocacy, research, and patient services.For more information contact:Alexa MooreVP of Development, National Organization for Rare Disorders617-934-6397 amoore@rarediseases.orgDerek GavinDirector of Development, National Organization for Rare Disorders617.249.7304 dgavin@rarediseases.org NORDsummit.org enicsRedefining Drug DiscDiscovery Through InnovationBRONZEALEXIONADDITIONAL SPONSORSAFFILIATEMedia Partners 2017 The National Organization for Rare Disorders (NORD ). All rights reserved.NORD is a registered trademark of the National Organization for Rare Disorders. Rare Diseases & Orphan Products Breakthrough Summit is a trademark of NORD. NORD is a registered 501(c)(3) charity organization. Please note that NORD provides this information for the benefit of the rare disease community. NORD is not a medical provider or healthcare facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Patients must rely on the personal and individualizedmedical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder.10

CONFERENCEPRICINGAdvantageRateStandardRateNORD Patient Organization Members 349 649 749Non-Profits/Patients/ Academics 399 699 799 1,699 2,099 2,199Government 499 499 599Students, Residents and Fellows 399 699 799Physicians, Pharmacists and Nurses(2-day Summit)* 499 699 799Industry (Pharma, Service Providers,Co-Pay Foundations) 2,199 2,499 2,599PricingNORD Corporate Council MembersExpired8/25/17Register by10/15/17RegisterOnsiteRegister at NORDSummit.org.*This price is offered to individuals attending the CME program offered by NORD during the conference. Please visitrarediseases.org/CME for more information.For additional pricing information, please contact NORDREG@MeetingExpectations.com.Registration fee includes continental breakfast, lunch, refreshments, wine and cheesereception and conference materials.Credit Card (Visa, MC, AMEX, Discover) or checks accepted. Please make checks (in U.S. fundsdrawn on a U.S. bank) payable to:National Organization for Rare Disorders, Inc. PLEASE NOTE: All advertised discountsDepartment 5430are taken from the full, standard rate.P.O. Box 4110, Woburn, MA 01888-4110VENUEMarriott Wardman Park2660 Woodley Rd NW,Washington, DC 20008Phone Reservations: 1-877-212-5752 (mention NORD)Please book your room by September 22, 2017.SUBSTITUTION AND CANCELLATIONScholarship ApplicationsNORD is pleased to provide patient organizations with scholarships to help with thecost of attending the Summit. Scholarships areawarded on a first-come, first-served, as-neededbasis with priority given to NORD patient organization members and students.Any cancellations received in writing on or before 14 days prior to the start date of the eventwill be refunded, less a 399 administrative charge. No refunds will be made after October 1,2017. Your registration may be transferred to another member of your organization up to 24hours in advance of the summit.To apply, please go to www.nordsummit.organd download the application.In case of a conference cancellation beyond our control*, you will receive a refund for yourconference registration fee only. NORD reserves the right to alter this program without priornotice. Please Note: Speakers and agenda are subject to change. In the event of a speakercancellation, every effort to find a suitable replacement will be made.Academics, researchers, industry, governmentagencies, health care professionals, patient organizations a

Featured FDA Speakers, Panelists & Moderators RICHARD A. MOSCICKI, M.D. Deputy Center Director for Science Operations, Center for Drug Evaluation and Research (CDER), FDA ROBERT TEMPLE, M.D. Deputy Center Director for Clinical Science, Center for Drug Evaluation and Research (CDER), FDA PETER MARKS, M.D., Ph.D.