TheAngelmanSyndromeConferencewillbeheldinHamburg,Germany

Location HyperionHotelHamburg Amsinckstraße39

20097Hamburghttps://www.h-hotels.com/de/hyperion/hotels/hyperion-hotel-hamburg

ScientificSymposium ThescientificsymposiumonFridayisplannedforexpertaudience

(researchersandscientists)butparentsarewelcometoattend.

Arrival Thursday,October11(evening),2018 Symposium Friday–Saturday,October12–13,2018 Departure Saturday,October13eveningor

Sunday,October14morning

ParentsDay:Saturday,October13,2018 Onsaturdayaprogramforgeneralaudienceisplanned.

6thInternational Scientific Conference 2018Germany

Executiveorganization:Angelmane.V.Germanywww.angelman.de 1/7

PatronJürgenVogelActor

Program *

Thursday,October11 19:00 MeetandGreetwithDinner

20:00 PlenaryLecture,chair:M.ScheffnerandH.Sitte(ASASAB)andB.Gerlach(Angelmane.V.Deutschland)

21:00 Get-together

Friday,October12(expertaudience)

8:30 Opening:BodoGerlach(Chair)andConnySchendler(bothAngelmane.V.)

8:40 Opening,chair:M.ScheffnerandH.Sitte

9:00 Plenaryspeaker:Y.Elgersma

Session1 BasicResearch 9:45 Speaker1:S.Chamberlain

10:15 Speaker2:B.Distel

10:45 Speaker3:S.Russo 11:15 Speaker4:B.Philpot 11:45 Lunchandscientificpostersession

Session2 Clinical(Transmitters/Genetics) 13:00 Speaker1:K.Egawa

13:30 Speaker2:C.Kannegießer-Leitner

14:00 Speaker3:B.Horsthemke

14:30 Coffee/teabreakandscientificpostersession

Session3 Pharma

15:15 Introduction,chair:M.ScheffnerandH.Sitte

15:20 Speaker1:EdWeeberforAGILISBiotherapeutics

15:50 Speaker2:MeghanMiller,ROCHEPharma

16:20 Speaker3:E.Schneider,IONISPharmaceuticals,Inc.(requested)

16:50 Speaker4:AmitRakhit,OVIDTherapeutics

17:30 Refreshmentsandscientificpostersession

19:30 DinnerandEveningevent:BoattourononeofHamburg‘srivers

openbar

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postersession

presentedbyparents

Program *

Saturday,October13(generalaudience)

8:15 Opening:B.GerlachandC.Schendler(bothAngelmane.V.)

Openingchair:M.ScheffnerandH.Sitte

8:20 PatronJürgenVogel,additionalInterviewswillbepossible

8:30 Speaker1:Y.Elgersma

9:00 Speaker2:K.EgawaorS.Chamberlain

9:30 Speaker3:C.Kannegießer-Leitner

10:00 Coffee/teabreak

10:30 Speaker4:E.WeeberforAGILISBiotherapeutics

11:00 Speaker5:MeghanMiller,ROCHEPharma

11:30 Speaker6:E.Schneider,IONISPharmaceuticals,Inc.(requested)

12:00 Speaker7:AmitRakhit,OVIDTherapeutics

12:30 Lunch

14:00 ChloeSimons,BoardofDirectors,AngelmanSyndromeTherapeuticsAustralia

14:15 TalksASAGrantWinner2018(S.Russo,B.Philpot)

15:30 RoundtableScientists/PharmasandASABoardMeeting

17:00 Departure

Prof.Dr.rer.nat.MartinScheffnerUniversityofKonstanz,Germany

Univ.Prof.Dr.HaraldSitteMedicalUniversityofVienna,Austria

BecausetheAngelmanSyndrome(AS)isararedisorder,ithasnotrececeivedthenecessaryattentiontomakeadifferenceinboththeacademicandpharmaceuticalworlds.Inthepasttenyears,thishas

changed,mainlyduetothespiritanddedicationoffamilieswithASchildrentobringtogetherscientistsfrombasicandclinicallyorientedresearchareas.Themostrecenthighlightofsuchga-theringshasbeenthebiennialinternationalscientificconferenceoftheAngelmanSyndromeAlliance(ASA),whichwillbeheldinGermanyin2018.IntheASAconference,distinguishedscientistsandclinicians/pediatriciansworkingondifferentAS-relatedaspectswillpresentanddiscussnewestfindingsanddevelopments,includingnoveltherapeuticstrategiesandtheirpotentialpitfalls.Suchdiscussi-onsareinvaluablefortheprogressofASresearchwithrespecttobothelucidationofthemechanismsunderlyingASandthedevelopmentoftreatmentstrategies.TheparticipationofparentorganizationsfromallovertheworldisanimportantfeatureoftheASAconference.TheregularcontactwithASfamiliesprovidesASresearcherswithaparticularmotivation.Also,thiswillensurethattheprogressmadeiscommunicatedtoASfamilies.

Astrongbasefornetworkingactivitiesisnecessarytostayuptodateandintouchwithalltheresearchersthathaveonecommongoalinmind:tounderstandhowtheAngelmanSyndromeessentiallyworks

andwhattodotoalleviateitssymptomsforthepatientsandtheirfamilies.Inthisregard,abiennialEuropeanmeetingisofutmostrelevanceforallresearchersinterestedintheAngelmanSyn-dromeandadjacentraredisorders.

ScientificAdvisoryBoard(SAB)

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TheGermanAngelmanAssociationisveryhappytoannouncethatJürgenVogel,oneofthemostfamousGermanactors,whoisalsoascreenwriter,comedian,filmproducerandsinger,hasagreedtobeourpatron.

*Subjecttochanges

Speakers

6thInternational Scientific ConferenceHamburg,Oct12-13,2018

MeghanMillerDiscoveryProjectLeader,RareDiseasesROCHEPharmaResearchandEarlyDevelopmentROCHEInnovationCenterBaselSwitzerland

TranslatingTherapiestoPatientsAngel-manSyndrome

BenPhilpotPhDPhilpotLab,UniversityofNorthCarolina,USA

Angelmansyndrometreatmentopportuni-tiesandclinicalbiomarkers

Dr.ChristelKannegießer-LeitnerResearchgroupAngelmanVereine.V.,Germany

CannabidiolextractasanewapproachintheanticonvulsivetreatmentoftheAngel-mansyndrome-basedontheneurotrans-mitterprofile,particularlyinconsiderationofGABAandglutamate

Prof.Dr.BenDistelPhDDepartmentofMedicalBiochemistry,AMCandDepartmentofNeuroscience,ErasmusMC,Netherlands

RegulationofthesubcellularlocalizationofUBE3A

AmitRakhit,MDChiefMedical&PortfolioOfficerOVIDTherapeuticsUSA

OvidTherapeutics:UpdateonAS

BernhardHorsthemkePhDProfessorandChairInstituteofHumanGeneticsEssen,Germany,UniversityHospital

Imprintingdefectsanddifferentialdia-gnoses

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Speakers

6thInternational Scientific ConferenceHamburg,Oct12-13,2018

YpeElgersma,PhDProfessorofMolecularNeuroscienceScientificdirectorENCOREExpertiseCentreforNeurodevelopmentalDisordersDepartmentofNeuroscience,ErasmusMC,Netherlands

MolecularmechanismsunderlyingASandinvestigatingtreatmentoptions

Dr.SilviaRussoPhDResponsabileSezioneGeneticaMalattieRare;Usso;IstitutoAuxologicoItaliano;LaboratorioBiologiaMolecolare,Italy

TaurineadministrationimprovesmotorandlearningdeficitsinASmousemodel

NaturalhistorystudyeffortsinAngelmanSyndromeandthescientificrationalefortherapywithantisenseoligonucleotides

AdenoAssociatedVirus-mediatedUBE3Ageneorproteinreplacementstrategies

StormyJ.ChamberlainChair,ScientificAdvisoryCommitteeAngelmanSyndromeFoundation,Inc.DepartmentofGeneticsandGenomeSciencesUConnHealth,USA

CellularphenotypesofhumanASiP-SC-derivedneurons

KiyoshiEgawaPediatricDepartment,HokkaidoUniversityGraduateSchoolofMedicine,Japan

GABA-mediatedinhibitatorydysfunctionanditspathophysiologicalcontributioninAngelmansyndrome

Prof.E.Weeber,PhDMolecularPharmacologyandPhysiologyCSOByrdAlzheimer’sInstitute,USAalsoforAGILISBiotherapeutics,USA

EugeneSchneiderVicePresident,ClinicalDevelopment,IONISPharmaceuticals,USA

requested

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Conference(paymenttoAngelmane.V./accountseebelow)

Ifyouhaveanyquestionsregardingbookings,paymentortheweekendpleasecontact:Mrs. ConnySchendlerphone +491792175898e-Mail conny.schendler@angelman.de

Contact

***Angelmankidsareverywelcomeattheconference.Angelmanchildrenwillhavefreeentry.Pleasebeawarethatnospecialchildrenprogramorassistancewillbeavailable.

Fees

Thursday–Saturday**: 315Euro*

Friday–Saturday**: 270Euro*

Fridayonly: 225Euro*

Saturdayonly: 195Euro*

SaturdayonlyformembersoftheAngelmane.V.***: 95Euro*

*Mealsanddrinksincluded.

Accommodation Hotel(Pleasebookthehoteldirectlyandmention

thepassword:ANGELMAN)

Singleaccommodation EUR129.90/night

Doubleaccommodation EUR149.90/night

PreferentialrateshavebeensecuredatHyperionHotelHamburg.

**ASAMemberswillreceiveadiscountofEUR100.00oneachconferenceparticipant.Thisdiscountislimitedto2ASAmembersofeachcountry.Additionalmemberswillpaythenormaldayrates.

Paymentsshouldbemadeviapaypal:conference@angelman.deReference:ASAConference/usingyourname

ortothefollowingbankaccount:SparkasseNeussAngelmane.V.DE03305500000093462885WELADEDNXXXASAConference/yourname

Wecannotacceptcreditordebitcardpayments.

BankAccountName

IBANBIC/SWIFTReference

Payment

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!1st September 2018!