2019 Translational Research Conference for the Management of NCLs

On November 7th & 8th, we will assemble a group of the world’s leading researchers in the Chicago area to discuss recent progress and future directions in translational research for all forms of Batten disease.
Batten disease refers to a family of progressive neurodegenerative disorders that are also known as the neuronal ceroid lipofuscinoses (NCLs). As a group, Batten disease is the most frequent neurodegenerative disease of childhood with a worldwide incidence of 1 per 14,000 to 1 per 67,000. All forms are monogenic, autosomal recessive disease that are characterized by the accumulation of lipopigments in lysosomes of cells throughout the body. They present early in childhood with a myriad of phenotypes including visual loss, seizures, cognitive impairment, language difficulty, and motor dysfunction. All forms are ultimately fatal.
Recent developments in translational research for these disorders have led to FDA approval of Brineura for treatment of CLN2 and gene therapy clinical trials for CLN6 and CLN3. The discovery of potential therapeutic approaches continue to lead us closer to more human clinical trials. Moreover, this conference aims to educate upon novel therapeutic strategies being explored outside the Batten disease research community that might have implications in the treatment of Batten disease.

Specific topics that will be covered at this conference include:
• Current Barriers and Strategies in Gene Therapy
• Current Topics in Small Molecule Drug Discovery and Delivery
• Emerging Genetic and Cellular Approaches
• Clinical Trial Design
• Emerging Technologies for Patient Reported Outcomes

Opportunities to sponsor the event are available and we ask that you carefully consider this opportunity. Sponsors will be recognized in printed and electronic means at the conference.

***Confirmed Speakers***
• Jodi McBride, PhD | Assistant Professor, Oregon National Primate Research Center
• Casey Maguire, PhD | Assistant Professor of Neurology, Harvard Medical School | Department of Nuerology, Massachusets General Hospital
• May Khanna, PhD | Assistant Professor, Department of Pharmacology, The University of Arizona
• Michelle Hastings, PhD | Associate Professor, Rosalind Franklin University of Medicine and Science

Opportunities to sponsor the event are available and we ask that you carefully consider this opportunity. Sponsors will be recognized in printed and electronic means at the conference.

Sponsorship levels include:
• Platinum: $5000+ (Includes registration for two attendees)
• Gold: $1500+ (Includes registration for two attendees)
• Silver: $500+ (Includes registration for one attendee)

If you plan to sponsor the event, please use the Donation registration in place of Conference Attendee registration.
We hope you join us as we gather families, scientists, and industry experts to discuss the latest in therapeutic developments and preclinical/clinical study design with cross cutting applications for multiple forms of Batten disease.



Chicago O’Hare International Airport (ORD)
Airport Phone: +1 800-832-6352Hotel direction: 16 miles NE
This hotel does not provide shuttle service.
• Alternate transportation: Via Limo; fee: 70 USD (one way) ;on request
• Estimated taxi fare: 40 USD (one way)
• Bus service, fee: 54 USD (one way)
Visit ORD airport website
Driving directions
O’Hare International Airport Take 294 South to I-88 West. Exit on Highland Ave & make right off exit to Butterfield Rd./ Rte 56. Turn right and hotel will be 1/4 mile down on right side Distance: 14 miles – Drive time: 25 minutes.

DuPage Airport (DPA)
Airport Phone: +1 630-584-2211
Hotel direction: 21 miles W
This hotel does not provide shuttle service.
• Estimated taxi fare: 41 USD (one way)
Visit DPA airport website
Driving directions
DuPage Airport to Westin Lombard Yorktown Center: Take Kautz Rd to IL-38 E/W Roosevelt Rd. Turn left onto IL-38 E/W Roosevelt Rd. Take Purnell Rd and Winfield Rd to IL-56 E/Butterfield Rd. Follow IL-56 E/Butterfield Rd to Fairfield Dr. Turn left onto Fairfield Dr. and bear right. Hotel is .8 mile on right.

Chicago Midway International Airport (MDW)
Hotel direction: 24 miles E
This hotel does not provide shuttle service.
• Estimated taxi fare: 40 USD (one way)
Visit MDW airport website
Driving directions
Midway International Airport (MDW): Take I-55 South to I-294 North. Follow signs to I-88 West. Follow to Exit 134 taking ramp right to Highland Ave. Turn right onto Highland Ave and take ramp right to IL-56/Butterfield Rd. Turn left onto S. Fairfield Ave and bear right. Hotel will be .7 miles down on the right.

If you have any questions prior to registration please contact Sarah or Heather Dainiak.

Please join and help us support a GALA hosted by Noah’s Hope/ Hope 4 Bridget Foundation the same weekend as the 2019 NCL Translational Research Conference for the Management of NCL’s
EVENT: The Fifth Season- Hope Shines on Batten
DATE: Saturday November 9, 2019
LOCATION: Chicago Marriott Oak Brook, 22nd Street, Oak Brook, IL, USA

Tickets available for A Fifth Season – Hope Shines on Batten. We hope you can join us on November 9th. You can support this event in several ways: buy tickets and come have a fun night and learn about Batten research updates, donate to the live/silent auctions, donate to the wine auction, become a sponsor! Please visit this site to attend or sponsor: https://e.givesmart.com/events/dOd/#page