Speakers: Tan Nyugen - FDA Office of Orphan Product Developoment, Jean Temeck - Regulatory Requirements for Pediatric Drug Development, Jill Heemskerk - A role for the NIH in Translational Research

Speakers: Ching Wang- Standard of Clinical Care for Spinal Muscular Atrophy, Jacqueline Jackson- International Spinal Muscular Atrophy Patient Registry, Richard Finkel- Parent Survey Data (not posted)

Existing US SMA Clinical Networks: Susan Iannaccone- AmSMART, Darryl De Vivo- The PNCR Network, Kathryn Swoboda- Project Cure SMA

Existing European SMA Clinical Networks: Enrico Bertini- Treat-NMD Overview, Michael Rose- NMD Registry of Outcome Measures

Susan Iannaccone- Natural History of SMA

Speakers: Petra Kaufmann- Issues in SMA Clinical Trials, Alfred Sandrock- Industry Perspective on SMA Clinical Trials (not posted)

Panel Discussion: Clinical Trial Design Issues; Panelists: Jean-Louis Abitbol, Wilson Bryan, Petra Kaufmann, Alfred Sandrock, Kathryn Swoboda, Ching Wang

Speakers: Agnes Saint Raymond (Video) Orphan Designation and Paediatric Initiative, Wilson W. Bryan- Regulatory Issues in SMA Clinical Trials

Speakers: Jill Heemskerk- Chemical Optimization of Indoprofen for the Treatment of SMA, Jon Tinsley- Use of a Drosophila SMN Model in a High Throughput Small Molecule Screen, Leo Liu- Identifying Novel SMA Drug Targets and Drug Candidates, Lou Barbato- Sodium Phenylbutyrate (NaPB) for the Potential Treatment of SMA (not posted), Mark Gurney- Families of SMA - deCODE – Clinical Candidate Selection, Paul Higgins- Identification of Tetracycline Compounds That Correct Defective SMA Splicing, Rebecca Pruss- Discovery and Development of TRO19622 for the treatment of SMA, Zuomei Li- Brain-Penetrant, Isotype-Selective HDAC Inhibitors, Sergey Paushkin- An Innovative and Integrated Approach to Identify Compounds (not posted)

Q&A session and Panel Discussion; Panelists: C. Frank Bennett, John McCall, Paul McGonigle, Thomas Meier, Robert Pacifici, Charlotte Sumner

Speakers: Kristin Krosschell- Functional Testing, Kathryn Swoboda- Electrophysiologic Outcomes CMAP and MUNE, Marion Main- Strength Testing and Walking in SMA 2&3, Richard Finkel- Type I-specific Measures (not posted), Anita Simonds- Outcomes Measurement: Pulmonary Assessment Type 1&2, Linda Hynan- Measuring Quality of Life in Patients with Spinal Muscular Atrophy, Louise Simard- Biomarkers for SMA Clinical Drug Trials

Q&A and Panel Discussion - Clinically Meaningful Endpoints for SMA Clinical Trials; Panelists: Wilson Bryan, Julaine Florence, David Jacoby, Kathryn Swoboda

Panel Discussion - Advancing SMA Drug Development: Challenges, Funding Priorities and Next Steps; Panelists: Petra Kaufmann, Edward Kaye, John Kissel, Langdon Miller, Tan Nguyen, Robert Pacifici

Dinner speaker presentation in Session 10 slides