H AT 6 T AXIA INVE ST IGAT ORS ME E T ING Glin z, PhD, Unive - PowerPoint PPT Presentation

H AT 6 T AXIA INVE ST IGAT ORS ME E T ING Gülin Öz, PhD, Unive rsity of Minne sota

DISCL AIME R  T he info rma tio n pro vide d b y spe a ke rs in a ny pre se nta tio n ma de a s pa rt o f the 2016 NAF Annua l Ata xia Co nfe re nc e is fo r info rma tio na l use o nly.  NAF e nc o ura g e s a ll a tte nde e s to c o nsult with the ir prima ry c a re pro vide r, ne uro lo g ist, o r o the r he a lth c a re pro vide r a b o ut a ny a dvic e , e xe rc ise , the ra pie s, me dic a tio n, tre a tme nt, nutritio na l supple me nt, o r re g ime n tha t ma y ha ve b e e n me ntio ne d a s pa rt o f a ny pre se nta tio n.  Pro duc ts o r se rvic e s me ntio ne d during the se pre se nta tio ns do e s no t imply e ndo rse me nt b y NAF .

PRE SE NT E R DISCL OSURE S  No r e lationships to disc lose or list

3 RD AIM - 2010

H AIM - 2012 4 T

H AIM - 2014 5 T

H AIM - 2016 6 T

AIM HIST ORY Numbe r of atte nde e s

170 atte nde e s from 12 c ountrie s on 4 c ontine nts

AIM HIST ORY Numbe r of sponsor s (othe r than NAF )

INDUST RY RE PRE SE NT AT ION: 13 7 sponsor s 6 r e gistr ants

AIM 2016 – GOAL S F ROM BASIC SCIE NCE T O CL INICAL T HE RAPE UT ICS 1. Ide ntify c ommon dise ase me c hanisms 2. E xplor e the r ape utic str ate gie s at the c linic al and basic sc ie nc e le ve ls 3. He lp e stablish futur e le ade r s of ataxia r e se ar c h by fac ilitating the involve me nt of young inve stigator s 4. Br ing tr aine e s into c ontac t with ataxia patie nts and the ir familie s

GOAL 1: DISE ASE ME CHANISMS he me 1: Ce r e be llar Cir c uitr y T he me 2: T he Biologic al basis of Ce r e be llar T De ge ne r ation he me 3: Ge nomic / Ge ne tic s Appr oac he s to T Ce r e be llar F unc tion and Ataxia

GOAL 2: T HE RAPE UT IC ST RAT E GIE S he me 4: T r anslational Re se ar c h T he me 5: Dr ug De ve lopme nt & T Clinic al T r ials 17/ 30 ta lks a bout the ra py c onc e pts!

HIGHL IGHT OF AIM & SAT E L L IT E ME E T INGS: Clinic al tr ial r e adine ss T ools to se le c t pa rtic ipa nts Outc ome me a sure s Na tura l history Me c ha nistic unde rsta nding

GOAL 3: E ST ABL ISH F UT URE L E ADE RS OF AT AXIA RE SE ARCH  18/ 30 le c tur e r s junior inve stigator  69 poste r s pr e se nte d Me gan Ke ise r , CHOP Jill F r ie dr ic h, UMN

GOAL 3: E ST ABL ISH F UT URE L E ADE RS OF AT AXIA RE SE ARCH  12 “hot c hair s” Vinc e nzo Ge nnar ino, Baylor Colle ge

GOAL 4: BRING T RAINE E S INT O CONT ACT WIT H PAT IE NT S  T hur sday dinne r spe ake r s patie nts & familie s  Poste r se ssion for patie nts & familie s

SCAL E S OF BRAIN http:/ / blog .visua l.ly/ visua lizing - the - sc a le - of- the - huma n- bra in/

WHAT RE AL L Y HAPPE NS We le ar n to spe ak e ac h othe r ’s language

WHAT RE AL L Y HAPPE NS We shape e ac h othe r ’s r e se ar c h to o raise a a scient ntist…

HOW YOU CAN HE L P Coor dination of Rar e Dise ase s at Sanfor d (CoRDS) • r e gistr y Clinic alT r ials.gov • NAF we bsite : • http:/ / www.ataxia.or g/ about/ how- you- c an- he lp.aspx

H AIM : 7 T F UT URE Apr il 2- 5, 2018 Philade lphia, PA

H AIM ST 6 T E E RING COMMIT T E E Chair - David L ync h, MD, PhD Co- c hair – Gulin Oz, PhD He nr y Paulson, MD, PhD Har r y Or r , PhD Ale xandr a Dur r , MD Al L a Spada, MD, PhD

NAF ST AF F & VOL UNT E E RS Mike Par e nt Sue Hage n David Gar c ia, photogr aphy

THANK YOU FOR SUPPORTING RESEARCH & INSPIRING US !