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March 3, 2016

5:00 pm - 8:00 pm       
Registration & Exhibits
Location: Foyer B

Poster Presentations & Welcome Dinner
Location: Regency C

March 4, 2016

7:30 am - 8:15 am
Registration & Exhibits
Location: Foyer B

Poster Presentations
Location: Regency C

Continental Breakfast
Location: Regency AB

Pompe Disease Newborn Screening

8:20 am - 8:40 am
Newborn screening laboratory methodology in Pompe Disease: Missouri Experience
Patrick Hopkins, BS,  Missouri Department of Health
Location: Regency C

8:40 am -  9:00 am
Newborn screening:  Rationale, Approval, Implementation, Logistical Issues of Short Term Follow-up
Sharmini Rogers, MBBS, MPH,  Chief, Newborn Screening Program, Missouri Department of Health
Location: Regency C

9:00 am - 9:30 am
Methodologic Considerations for Pompe Disease Newborn Screening
Michael Gelb, PhD, University of Washington
Location: Regency C

9:30 am – 10:00 am 
Care and Outcomes of Infantile Cases Using Enzyme Replacement Therapy
Yin-Hsiu Chien, MD PhD, National Taiwan University
Location: Regency C

10:00 am - 10:30 am
Best Care Guidelines in Later-onset Pompe Disease: Consensus versus Controversy
Tahseen Mozaffar, MBBS, University of California, Irvine
Location: Regency C

10:30 am - 10:45 am
Break & Exhibits
Location: Foyer B

10:45 am - 11:15 am
Beyond ERT:  New Treatments for Pompe Disease
Barry Byrne, MD PhD, University of Florida
Location: Regency C

11:15 am - 11:45 am
Cost Effectiveness Analysis of Pompe Disease NBS
Lisa Prosser, PhD, University of Michigan
Location: Regency C

11:45 pm - 12:00 pm
Questions for Pompe Disease Speakers
Location: Regency C

12:00 pm - 1:00 pm
Location: Regency AB

Duchenne Muscular Dystrophy Newborn Screening

1:00 pm - 1:30 pm
DMD-NBS Now versus Then:  Improving Laboratory Methods and Screening Outcomes?
Veronica Wiley, PhD, University of Sydney
Location: Regency C

1:30 pm - 2:00 pm
Newborn Screening in China and the Introduction of DMD NBS
Zhengyan Zhao, MD, Zhejiang University
Location: Regency C

2:00 pm - 3:00 pm 
New Treatments for DMD: Earlier Treatment Leads to Better Outcomes
Update on SMA Treatment Trials - Late Breaking News
Jerry Mendell, MD, PhD, Ohio State University
Location: Regency C

3:00 pm - 3:20 pm
DMD-NBS Follow-up: What Should Short Term and Long-term Follow-up Care Look Like?
Anne Connolly, MD, Washington University School of Medicine
Location: Regency C

3:20 pm - 3:30 pm 
Questions for DMD Speakers
Location: Regency C

3:30 pm - 3:45 pm

Break & Exhibits
Location: Foyer B

Assuring that NBS Programs Meet Public Health Needs

3:45 pm - 4:15 pm
Challenges for Implementation of Neuromuscular NBS in the US
Jelili Ojodu, MPH, Association of Public Health Laboratories
Location: Regency C

4:15 pm - 4:45 pm 
Workforce Challenges in the Implementation of Neuromuscular NBS
Peter Kang, MD, University of Florida
Location: Regency C

4:45 pm - 5:15 pm
Ethical Issues with Neuromuscular Disease Newborn Screening
Laine Ross, MD, PhD, University of Chicago
Location: Regency C

6:00 pm - 8:00 pm
Dinner & Brief Review of Clinical Outcomes Registries in DMD and Pompe Disease
Location: Regency AB

Poster Viewing
Location: Regency C

March 5th, 2016

7:30 am - 8:30 am
Location: Regency AB

8:30 am - 9:30 am  
Panel Discussion: Introduction to Clinical Outcomes Registries
Discussion Led by Registry Experts
Location: Regency AB

9:30 am - 10:30 am
Roundtable of Pompe Disease NBS Clinical Follow-up[ Providers: Case Presentations and Discussion of Protocol and Registry
Clinical Providers from Missouri and New York
Location: Regency AB

10:30 am - 11:15 am
Working Groups:  Creating Draft Neuromuscular NBS Short- and Long-term Follow-up Protocols for Pompe Disease and DMD
Steering Committee and Clinical Providers to Facilitate
Location: Regency AB

11:15 am - 12:00 pm

Discussion of Next Steps
Steering Committee to Facilitate
Location: Regency AB

Register for this event!