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

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H AT

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

3RD AIM - 2010

4T

H AIM - 2012

5T

H AIM - 2014

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H AIM - 2016

AIM HIST ORY

Numbe r

• f atte nde e s

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

AIM HIST ORY

Numbe r

• f 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

T 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 De ge ne r ation T he me 3: Ge nomic / Ge ne tic s Appr

• ac he s to

Ce r e be llar F unc tion and Ataxia

GOAL 2: T HE RAPE UT IC ST RAT E GIE S

T he me 4: T r anslational Re se ar c h T he me 5: Dr ug De ve lopme nt & 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

Me c ha nistic unde rsta nding Na tura l history Outc ome me a sure s T

• ols to se le c t pa rtic ipa nts

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

 12 “hot c hair

s”

GOAL 3: E ST ABL ISH F UT URE L E ADE RS OF AT AXIA RE SE ARCH

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

• the 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

• 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

F UT URE : 7T

H AIM Apr il 2- 5, 2018 Philade lphia, PA

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H AIM ST

E E RING COMMIT T E E

Chair

• David L

ync h, MD, PhD Al L a Spada, MD, PhD Har r y Or r , PhD Ale xandr a Dur r , MD He nr y Paulson, MD, PhD Co- c hair – Gulin Oz, PhD

NAF ST AF F & VOL UNT E E RS

Mike Par e nt David Gar c ia, photogr aphy Sue Hage n

THANK YOU

FOR SUPPORTING RESEARCH & INSPIRING US!