Sanjay Bidichandani, MBBS, PhD Vice President for Research

1. Translational Research “Preclinical Drug Development” Target Identification Drug Screening “Proof-of-Principle” Testing in Animals Clinical Research Including Trials Basic Research Sanjay Bidichandani, MBBS, PhD Vice President for Research

2. MDA’s Research Portfolio Total ~300 grants ~$100 M Annually ~100 new grants ~$40 M • Medical Advisory Committee • Scientific Advisory Committee • Translational Res Advisory Committee • Ad hoc peer-review

3. MDA’s Research Portfolio Muscular Dystrophies Duchenne Becker Limb-Girdle Facioscapulohumeral Congenital Oculopharyngeal Distal Emery-Dreifuss Myotonic dystrophy Motor Neuron Disease Amyotrophic Lateral Sclerosis Spinal Muscular Atrophy Spinal Bulbar Muscular Atrophy Inflammatory Myopathies Polymyositis Dermatomyositis Inclusion Body Myositis Neuromuscular Junction Diseases Myasthenia Gravis Lambert-Eaton Syndrome Congenital Myasthenic Syndromes Endocrine Myopathies Hyperthyroid Myopathy Hyopthyroid Myopathy Diseases of Peripheral Nerve Charcot-Marie-Tooth Disease Friedreich Ataxia Dejerine-Sottas Disease Metabolic Myopathies Phosphorylase Deficiency Acid Maltase Disease Phosphofructokinase Deficiency Debrancher Enzyme Deficiency Mitochondrial Myopathy Carnitine Deficiency CarnitinePalmitylTransferase Deficiency Phosphoglycerate Kinase Deficiency PhosphoglycerateMutase Deficiency Lactate Dehydrogenase Deficiency MyoadenylateDeaminase Deficiency Other Myopathies MyotoniaCongenita ParamyotoniaCongenita Central Core Disease Nemaline Myopathy Myotubular Myopathy Periodic Paralysis

4. MDA’s Research Portfolio Translational Research “Preclinical Drug Development” Drug Screening Target Identification “Proof-of-Principle” Testing in Animals Clinical Research Including Trials Basic Research Basic Research Program Basic Research Program Translational / Basic Research Program Translational Translational / other $60.8 $14.2 $7.2 $3.7 $4.8 Active projects (million)

5. MDA’s Research Portfolio ** FY10 / non-ARRA

6. MDA VENTURE PHILANTHROPY Turning Research into Treatments

7. 2 yrs 1yr 1.5-2yrs 1 yr 1 yr 2 yr 2-3 yrs Hit ID Lead Optim. Target ID Pre- IND P3 P1 Reg P2 Can Risk Cost MDA’s Drug Development Program Translational research MDA Biotech Academia Big Pharma

9. MDA’s Recent Advances In Therapy Development SMA Repligen Friedreich ataxia Repligen Friedreich ataxia Repligen Duchenne / Becker Askelepios ReveraGen Duchenne / Becker Duchenne PTC LGMD2d Mendell Miller ALS (SOD1) IIb I/IIa PreC PoC

10. …and that’s a good thing… “It’s a great time to be a mouse with a neuromuscular disease!” John Porter, Ph.D. (NIH / NINDS)

11. Challenges How to safely and effectively extend these successes to humans? Who is going to fill the void between biotechs funded by foundations & fed - and - big pharma?

12. Smart partnership….

13. Finding Treatments: Smart Partnership • Leveraging through MDA’s drug development program • Federal govt & other non-profits • Partnership with Pharma and Biotech companies • Venture capitalists

14. Enhancing Research through Communication & Collaboration

15. MDA Symposium Series - 2012 MDA Neuron Symposium Role of glia in motor neuron degeneration in ALS MDA Muscle Symposium Newborn screening for Duchenne muscular dystrophy MDA Translational Symposia Encouraging technology transfer from academia Gene therapy for neuromuscular disease (with AFM)

16. Getting a head start…

17. Clinics & Clinical Research Networks MDA Clinic MDA ALS Clinic Duchenne research network ALS research network

18. Investment in Training Researchers…. Currently = 40 Trainees (~$7 million) Historically = 377 trainees (~ $48 million) • Basic researchers (DG) • Clinical researchers (CRTG)

19. MVP: Program Review Zero trainees in translational projects!

20. Academia Industry Bridge to Industry (B2I) • B2I postdoctoral fellowships • Training future NMD translational researchers

21. Future Muscle Disease Researchers