A New Era of Medicine Is Upon Us

A NEW ERA OF MEDICINE IS UPON US

SUMMER 2019 NASDAQ: SRPT FORWARD-LOOKING STATEMENTS

This presentation contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our opportunities in the rare disease space; potential solutions and market opportunities with our RNA technologies, gene therapy and gene editing; the potential of gene therapy’s applicability across disease; the potential benefits of our technologies and scientific approaches, including the potential of RNA-targeted medicine to increase or decrease production of a protein involved in a disease, the potential of exon skipping to allow for production of an internally truncated but functional dystrophin protein, the potential benefits of PMO and PPMO, including PPMO’s potential to greatly increase cell penetration, lead to more efficient dosing and deliver to unique muscle types; the potential benefits of MHCK7, AAVrh74, SR2, SR3 and β-SARCOGLYCAN; the expectation to have 3 RNA therapies on the U.S. market by 2020, serving ~30% of the DMD community; our plans for the future and expected milestones, including developing PPMOs that can serve 43% of DMD, a target regulatory approval for golodirsen in 2019 and for casimersen in 2020; our goal to identify 2 gene therapy product candidates per year; the potential of our LGMD portfolio to generate a steady stream of gene therapy candidates; the potential benefits of iCELLis adherent mammalian manufacturing technology; our forecast curve for gene therapy; our sustainable model for transformative gene therapies, including having gene therapy manufacturing capacity in 2020 and an access and reimbursement plan in 2021; our expected development horizon of 3.5 years; the estimated number of patients suffering from DMD, LGMD, CMT, MPS IIIA and pompe; and our goal to have 40 programs and the estimated epidemiology associated with the different diseases. These forward‐looking statements involve risks and uncertainties, many of which are beyond our control and are based on our current beliefs, expectations and assumptions regarding our business. Actual results and financial condition could materially differ from those stated or implied by these forward‐looking statements as a result of such risks and uncertainties and could materially and adversely affect our business, results of operations and trading price. Potential known risk factors include, among others, the following: our data for our different programs, including golodirsen, casimersen, micro-dystrophin and LGMD may not be sufficient for obtaining regulatory approval; our product candidates, including those with strategic partners, may not result in viable treatments suitable for commercialization due to a variety of reasons including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; even if our programs result in new commercialized products, we may not achieve any significant revenues from the sale of such products; success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; if the actual number of patients suffering from DMD, LGMD, MPS IIIA, CMT and/or pompe is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; various factors may decrease the market size of our product and product candidates, including the severity of the disease, patient demographics and the response of patients’ immune systems to our product candidates; our dependence on our manufacturers to fulfill our needs for our clinical trials and commercial supply, including any inability on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet product demand, may impair the availability of products to successfully support various programs, including research and development and the potential commercialization of our gene therapy product candidates; we may not be able to successfully scale up manufacturing of our product candidates in sufficient quality and quantity or within sufficient timelines; current reimbursement models may not accommodate the unique factors of our gene therapy product candidates; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, for various reasons including possible limitations of our financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office; and those risks identified under the heading “Risk Factors” in Sarepta’s 2018 Annual Report on Form 10‐K or and most recent Quarterly Report on Form 10‐Q filed with the Securities and Exchange Commission (SEC) and in its other SEC filings. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's filings with the SEC. We caution investors not to place considerable reliance on the forward‐looking statements contained in this presentation. The forward‐looking statements in this presentation are made as of the date of this presentation only and, other than as required under applicable law, Sarepta does not undertake any obligation to publicly update its forward‐looking statements.

3 7,000 Rare Diseases ~80% are single gene mutations

And only 5% of rare diseases currently have treatments

© SAREPTA THERAPEUTICS, INC. 2019. ALL RIGHTS RESERVED. 4 Rare diseases are responsible for % 400MRare disease patients w/w % 30 35 of children of deaths with a rare disease 50% in the first year of life* will never reach their Children* 5th birthday* ..AND THE STATISTICS SOBERING

EVERY DAY Duchenne takes the life of a child

*http://www.fromhopetocures.org/fighting-rare-diseases

6 SAREPTA IS LEADING THIS REVOLUTION

Armed with the most advanced science in genetic medicine, we are in a daily race to rescue lives otherwise stolen by rare disease. At Sarepta, every day is another 24 hours to stand up for patients, advance technology, challenge convention and drag tomorrow into today.

RNA GENE THERAPY GENE EDITING

PMO PPMO Two approaches to DMD; Six forms of Limb-girdle muscular dystrophy (LGMD); Duchenne Muscular Charcot-Marie-Tooth (CMT) disease; Mucopolysaccharidosis type IIIA (MPS IIIA); DMD Dystrophy (DMD) Pompe disease/CNS 11 12 1 PROGRAMS PROGRAMS PROGRAM

9 AN ELEGANT RNA PRECISION GENETIC MEDICINE APPROACH

RNA Pre-mRNA mRNA Ribosome translates Protein is Transcription spliced into leaves the mRNA and assembles transported to begins mature mRNA nucleus the protein perform its function

1 2 3 4 5

SAREPTA’S RNA-TARGETED THERAPEUTIC

RNA-targeted Medicine - Designed to increase or decrease production of a protein involved in a disease

PHOSPHORODIAMIDATE PEPTIDE PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER MORPHOLINO OLIGOMER (PMO) CHEMISTRY (PPMO) CHEMISTRY

Specificity: Enhanced affinity for targeting Enhances PMO pre-mRNA for precise binding to the selected RNA target • Same precision genetic Stability: Highly resistant to degradation medicine backbone by enzymes • Conjugated peptide greatly increases cell penetration Versatility: Ability to rapidly design and • Could potentially lead to more construct drug candidates that are specific efficient dosing for patients for human or pathogen RNA; and target specific tissues • Able to deliver PMOs to unique muscle types Safety: Built upon a charge-neutral backbone, (e.g., heart) not able to be which may be reflected in tolerability reached by PMOs (e.g., heart). © SAREPTA THERAPEUTICS, INC. 2019. ALL RIGHTS RESERVED. 11 PMO TECHNOLOGY FOR DUCHENNE MUSCULAR DYSTROPHY

PROBLEM: Duchenne is caused by a genetic mutation that prevents the body from producing dystrophin, a protein the muscles need to work properly.

PRECISION GENETIC Phosphorodiamidate Morpholino Oligomers (PMOs) are designed in precise sequence, bind to MEDICINE: the target, and direct the body to make a functional form of the dystrophin protein.

EXON SKIPPING:

46 47 48 49 51 52 53 54 55 56

Dystrophin Gene

PROBLEM: Duchenne is caused by a genetic mutation that prevents the body from producing dystrophin, a protein the muscles need to work properly.

EXON SKIPPING:

46 47 48 49 52 53 54 55 56 57

Dystrophin Gene

If successful, Sarepta will have three RNA-therapies on the U.S. market by 2020, serving ~30% of the DMD community

2016 2019 2020 Future

ETEPLIRSEN* GOLODIRSEN CASIMERSEN • PPMOs FOR DMD SRP-4053 SRP-4045 • OTHER DISEASE TARGETS Approved to treat patients Being developed to treat patients Being developed to treat patients with genetic mutations that are with genetic mutations that are with genetic mutations that are amenable to exon 51 skipping amenable to exon 53 skipping amenable to exon 45 skipping (13% of DMD population). (8% of DMD population). NDA-stage (8% of DMD population). therapy for patients.

Exon skipping is intended to allow for production of an internally truncated but functional dystrophin protein

15 SAREPTA’S GENE THERAPY ENGINE AT WORK: NOW, AND INTO THE FUTURE

PROGRAM DISCOVERY PRECLINICAL CLINICAL COMMERCIAL SRP-9001 Micro-dystrophin SRP-9003 (LGMD2E β-sarcoglycan) SRP-9004 (LGMD2D α-sarcoglycan) SRP-9005 (LGMD2C γ-sarcoglycan) MYO-201 (LGMD2B Dysferlin) SRP-9006 (LGMD2L Anoctamin 5) Calpain 3 (LGMD2A) SRP-9002 (MPS IIIA) (Lysogene) GALGT2 (Nationwide Children’s) Micro-dystrophin (Genethon) Neurotrophin 3 (CMT1A) (Nationwide Children’s) Pompe Disease (Lacerta) CNS-1 (Lacerta) CNS-2 (Lacerta)

2 TARGETS PER YEAR - NEW GENE THERAPY CENTER OF EXCELLENCE

VECTOR Must deliver the transgene to target cells with minimal immune response PROMOTER Drives expression in intended tissues

TRANSGENE Produces a functioning version of the protein of interest

EARLY CLINICAL RESULTS NINE-MONTH CLINICAL RESULTS MICRO-DYSTROPHIN GENE MICRO-DYSTROPHIN GENE THERAPY FOR DMD THERAPY FOR DMD* VECTOR – AVERAGE CHANGE FROM BASELINE*** AAVrh74 • Provides systemic delivery to muscle NSAA TIME TO RISE cells, including the 81% 96% heart and skeletal muscle Expression of micro- Expression of dystrophin in muscle micro-dystrophin measured • Low level of pre-existing 1 1 PROMOTER .8 immunity 6.5 fibers by signal intensity POINT IMPROVEMENT SECOND IMPROVEMENT MHCK7 • Optimized for desired skeletal and cardiac muscle expression levels TRANSGENE • 120% expression in cardiac muscle vs skeletal 4 STAIRS UP 100 M 96% 64% MICRO-DYSTROPHIN muscle2 Expression of Reduction of • Designed to generate a micro-dystrophin creatine kinase1 functional micro-dystrophin 1.2 7.95 measured by SECOND IMPROVEMENT SECOND IMPROVEMENT • Includes SR2 and SR3 - Western blot**,1 essential for muscle force

1. Clinical Update: Micro-dystrophin Study-101 - March 25, 2019 - Micro-dystrophin Gene Therapy Update Conference Call - Sarepta Therapeutics Data on File 2. Potter et al. Functional and Histological Improvements Comparing 4 Micro-dystrophin Constructs in the mdx Mouse Model of DMD. ASGCT 2019 ClinicalTrials.gov Identifier: NCT03375164 2. Potter et al. Functional and Histological Improvements Comparing 4 Micro-dystrophin Constructs in the Sarepta Therapeutics Data on File mdx Mouse Model of DMD. AIM 2019

© SAREPTA THERAPEUTICS, INC. 2019. ALL RIGHTS RESERVED. *Data from the 4 patients dosed in Study NCT03375164 18 **NCH Western blot method ***North Star Ambulatory Assessment (NSAA), Time to Rise, 4 Stairs Up, and 100M SAREPTA’S GENE THERAPY ENGINE AT WORK – LGMD 2E CONSISTENT RESULTS IN NEW THERAPEUTIC AREA

PRELIMINARY CLINICAL RESULTS β-SARCOGLYCAN GENE THERAPY FOR LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2E*,1 VECTOR AAVrh74 51% 47% Expression of Expression of β-sarcoglycan in β-sarcoglycan measured muscle fibers by signal intensity PROMOTER MHCK7

TRANSGENE 36% 90% β-SARCOGLYCAN Expression of Reduction of β-sarcoglycan creatine kinase measured by Western blot**

*Data from the 3 patients dosed in Study NCT03652259 **NCH Western blot method 1. http://investorrelations.sarepta.com/news-releases/news-release-details/ sarepta-therapeutics-announces-positive-and-robust-expression

OPPORTUNITY TO GENERATE AAVrh74 A STEADY STREAM OF MHCK7 GENE THERAPY CANDIDATES β-sarcoglycan LGMD PORTFOLIO LGMD2E

AAVrh74 AAVrh74 AAVrh74 AAVrh74 AAVrh74

tMCK MHCK7 MHCK7 tMCK tMCK

α-sarcoglycan γ-sarcoglycan Dysferlin Anoctamin Calpain 3

LGMD2D LGMD2B LGMD2C LGMD2L LGMD2A

DUCHENNE LIMB-GIRDLE MUCOPOLYSACCHARIDOSIS MUSCULAR DYSTROPHY MUSCULAR DYSTROPHY

CHARCOT-MARIE-TOOTH POMPE DISEASE DISEASE

22 SAREPTA’S MANUFACTURING EXPERTISE

Process Development Analytical Development / Quality Control

Next-Generation Quality Assurance Technology

Supply Chain Drug Product

A deliberate and HYPERstackstrategic moveadherent from HYPERstack mammalianadherent mammalian to iCELLis® adherent mammalian

iCELLis® adherent mammalian

BENEFITS A deliberate and strategic move from HYPERstack adherent mammalian to iCELLis® adherent mammalian • Mitigated Risk • Increased Speed • Expanded Scale • Improved Cost Efficiency

27 CHANGING LIVES WITH JUST ONE DOSE: FAR-REACHING IMPACT FOR 21ST CENTURY MEDICINE

CHRONIC ONE-TIME

Life-long administration Single administration

High hospital costs Lower hospital costs

Significant financial burden on system Lower burden on system (vs. life long therapies)

So, what does a one-time therapy model look like?

CHRONIC DOSING MODEL . . .

Q1 Q2 Q3 Q4 Q5 Q6 Q7 Q8 Q9 Q10 Q11 Q12 Q13 Q14 Q15 Q16 Q17 Q18 Q19 Q20

Product 1 Product 2 Product 3 Product 4 Product 5 Product 6 Product 7 Product 8 Product 9 Product 10

ONE-TIME TREATMENT MODEL . . .

Q1 Q2 Q3 Q4 Q5 Q6 Q7 Q8 Q9 Q10 Q11 Q12 Q13 Q14 Q15 Q16 Q17 Q18 Q19 Q20

Product 1 Product 2 Product 3 Product 4 Product 5 Product 6 Product 7 Product 8 Product 9 Product 10

CREATING A VIABLE BUSINESS MODEL FOR GENE THERAPY

Q1 Q2 Q3 Q4 Q5 Q6 Q7 Q8 Q9 Q10 Q11 Q12 Q13 Q14 Q15 Q16 Q17 Q18 Q19 Q20

Product 1 Product 2 Product 3 Product 4 Product 5 Product 6 Product 7 Product 8 Product 9 Product 10

DISCOVERY ENGINE Differentiated technologies STEADY STREAM OF 3.5 unmatched in biotech GENE THERAPIES YEARS (from discovery)

MANUFACTURING READY IN The most manufacturing capacity globally 2020 FULFILL CLINICAL & COMMECIAL NEEDS

ACCESS AND READY IN REIMBURSEMENT Working with key 2021 stakeholders to bring medicines to patients PATIENTS

33 TODAY’S OPPORTUNITY: 24 PROGRAMS IN DEVELOPMENT

INHERITANCE DMD is inherited in an X-linked recessive pattern.1 Genetic Root of Disease One or more mutations in the gene SYMPTOMS that codes for dystrophin3 Muscle weakness becomes increasingly noticeable by age 3 to 5, and most patients use a wheelchair by the time they are 11. During adolescence, cardiac and respiratory muscle deterioration lead to Missing Protein Dystrophin3 serious, life-threatening complications. 1,2 Cellular Alteration Instability of skeletal and cardiac muscle POPULATION cell membranes3 DMD affect approximately 1 in 3,500-5,000 males born worldwide.3 Tissue Deterioration Muscle deterioration and replacement with fatty and fibrotic tissue1 TREATMENT Exon-skipping drugs are available for patients with certain dystrophin mutations, broadly applicable gene therapy in Function Loss Typically loss of ambulation in early teens 4 development. and progressive pulmonary and cardiac complications leading to mortality in late 20s.2

© SAREPTA THERAPEUTICS, INC. 2019. ALL RIGHTS RESERVED. 1.Hoffman EP, Brown RH, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987;51:919-928; 2. Passamano L, Taglia A, et al. Improvement of survival in Duchenne Muscular 35 Dystrophy: retrospective analysis of 835 patients. Acta Myologica. 2012;31(1): 121-125; 3. Duchenne and Becker muscular dystrophy. Genetics Home Reference 2019. https://ghr.nlm.nih.gov/condition/duchenne-and- becker-muscular-dystrophy#genes Accessed May 2019 4. Duchenne Muscular Dystrophy. Muscular Dystrophy Association. https://www.mda.org/disease/duchenne-muscular-dystrophy Accessed May 2019. LIMB-GIRDLE MUSCULAR DYSTROPHY (LGMD)

INHERITANCE The LGMDs are a group of genetically heterogeneous, autosomal Genetic Root of Disease Broad range of mutations in various genes inherited (recessive more common than dominant) muscular responsible for protein production3 dystrophies with a childhood to adult onset. 1 Missing Proteins Depends on specific subtype, such as proteins SYMPTOMS involved in the dystrophin associated protein Individuals may first notice a problem when they begin to walk with a “waddling” gait because of weakness of the hip and leg complex (DAPC), sarcolemma, dystroglycan muscles. They may have trouble getting out of chairs, rising complex as well as intracellular proteins2 from a toilet seat or climbing stairs. As this weakness progresses, the person may require the use of assistive mobility devices.3 Cellular Alteration These critical proteins are responsible for muscle function, regulation and repair3 POPULATION Approximate global prevalence of LGMDs as a group is 1.63 per 100,000 (Prevalence estimates range from 0.56 to 5.75 Affected muscles Affects skeletal muscle and in some cases per 100,000).1 Over 30 subtypes exist.2 Both genders are affects cardiac and/or diaphragm2,3,4 affected equally.3 Function Loss Progressive weakness and wasting of hip or TREATMENT 1 No cause-specific treatment is available for any of the LGMD shoulder girdle muscles ; in some cases subtypes. 2 cardiac abnormalities and respiratory decline3

© SAREPTA THERAPEUTICS, INC. 2019. ALL RIGHTS RESERVED. 1. Liewluck T, Milone M. Untangling the complexity of limb-girdle muscular dystrophies. Muscle Nerve. 2018;58(2):167-177. 36 2. Murphy AP, Straub V. The Classification, Natural History and Treatment of the Limb Girdle Muscular Dystrophies. J Neuromuscular Diseases. 2015;2(s2):S7-S19. 3. Limb-girdle muscular dystrophy (LGMD). Muscular Dystrophy Association. https://www.mda.org/disease/limb-girdle-muscular-dystrophy Accessed May 2019. MUCOPOLYSACCHARIDOSIS TYPE IIIA (MPS IIIA)

INHERITANCE Autosomal recessive, neurodegenerative lysosomal storage disease caused by mutations in the N-sulfoglucosamine sulfohydrolase (SGSH) Genetic Root of Disease Mutation in the SGSH gene4 gene, which encodes an enzyme called Heparan-N-sulfamidase necessary for heparan sulfate (HS) recycling in cells. 1,2 Deficient enzyme Heparan-N-sulfamidase5 SYMPTOMS Signs and symptoms usually become apparent in early childhood Cellular Alteration Build up of HS in cells5 and include speech problems, developmental delays, challenging behaviors, extreme hyperactivity and poor sleep.3 Tissue Deterioration Neurodegeneration5 POPULATION MPS IIIA has a worldwide incidence 0.27-2.89 per 100,000 births.1 Function Losses Significant cognitive impairment, loss of speech, death within second decade of life3 TREATMENT No approved product indicated to treat MPS IIIA. 3

1. Heron B. Incidence and Natural History of Mucopolysaccharidosis Type III in France and Comparison with United Kingdom and Greece. American Journal of Medical Genetics Part A. 2011; 155A(1):58-68. 2. NIH. Genetics Home Reference. SGSH gene. https://ghr.nlm.nih.gov/gene/SGSH#normalfunction Accessed May 2019. 3. NIH. Genetic and Rare Diseases Information Center. Mucopolysaccharidosis type IIIA. https://rarediseases.info.nih.gov/diseases/7071/mucopolysaccharidosis-type-iiia Accessed May 2019. 4. NIH. Genetics Home Reference. Mucopolysaccharidosis type III. https://ghr.nlm.nih.gov/condition/mucopolysaccharidosis-type-iii Accessed May 2019. 5. Muenzer J. Overview of the mucopolysaccharidoses. Rheumatology (Oxford). 2011;50(suppl 5):v4-12.

INHERITANCE 1 CMT1A is an autosomal dominant disease resulting from the Genetic Root of Disease Duplication of PMP22 gene duplication of the PMP22 gene.1 Protein Accumulation Aggresomes build up in the Schwann cell SYMPTOMS impairing its ability to survive 7 CMT1A symptoms involve muscle wasting leading to foot drop, balance difficulties and muscle weakness which can progress to affect motor skills limiting ability to walk and use hands. 2,3 Cellular Alteration Damage to Schwann cells leads to demyelination 1 POPULATION CMT is the most common inherited neuromuscular Tissue Deterioration Lack of myelin coating leads to disorder, affecting over 2.8 million people worldwide.3 1 CMT1A is estimated to be >30% of all CMT cases.4,5,6 degeneration of peripheral nerves

TREATMENT Function Loss Limited to no ability of the nerves to 1 No approved product indicated to treat CMT1A.3 communicate with their targets (muscles)

1. Charcot-Marie-Tooth Disease Fact Sheet. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Charcot-Marie-Tooth-Disease-Fact-Sheet Accessed May 2019. 2. Charcot-Marie-Tooth disease type 1A Fact Sheet. National Center for Advancing Translational Sciences. Genetic and Rare Disease Information Center. https://rarediseases.info.nih.gov/diseases/1245/charcot-marie-tooth-disease-type-1a Accessed May 2019. 3. What is CMT? Charcot-Marie-Tooth Association. https://www.cmtausa.org/understanding-cmt/what-is-cmt/ Accessed May 2019. 4. Types and Causes. Charcot-Marie-Tooth Association. https://www.cmtausa.org/understanding-cmt/types-and-causes/ Accessed May 2019. 5. Pareyson D, Saveri P, Pisciotta C. New developments in Charcot-Marie-Tooth neuropathy and related diseases. Curr Opin Neurol. 2017;30(5):471-480. 6. Saporta M, Sottile S. Charcot Marie Tooth (CMT) Subtypes and Genetic Testing Strategies. Ann Neurol. 2011; 69(1):22-33. 7. Niemann A, Berger P, Suter U. Pathomechanisms of Mutant Proteins in Charcot-Marie-Tooth Disease. NeuroMol Med. 2006; 8(1-2):217-242.

INHERITANCE Genetic Root of Disease Mutations in GAA gene2 Pompe is inherited in an autosomal recessive pattern.1 It is caused by a mutation in the gene that codes for the enzyme acid alpha- glucosidase (GAA), responsible for metabolizing glycogen in lysosomes. Deficient enzyme Acid alpha-glucosidase1 Over 300 different mutations in the GAA gene identified, disease varies widely in terms of age of onset (early or late) and severity.2 Cellular Alteration Build-up of glycogen in lysosomes1 SYMPTOMS Infantile onset Generally begins in the first months of life, includes Tissue Deterioration Accumulation in cardiac and skeletal feeding problems, cardiac and skeletal muscle weakness and frequent muscle and CNS tissue1 respiratory infections.1,2 Late onset (juvenile/adult) Primary symptom is muscle Infantile onset : Fatal in infants before weakness progressing to respiratory weakness along with feeding Function Loss 2 and swallowing complications.1,2 age 1.

POPULATION Late onset: Muscle weakness leading to Pompe has a worldwide incidence 1 in 40,000 births; respiratory weakness and fatal respiratory affects both sexes equally.2 failure (over a course of years).2 TREATMENT Approved enzyme replacement therapies are the standard of care.2

© SAREPTA THERAPEUTICS, INC. 2019. ALL RIGHTS RESERVED. 1. Kishnani P, Steiner R. Pompe disease diagnosis and management guideline. Genet Med. 2006; 8(5):267-288 39 2. Pompe Disease Information Page. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/Disorders/All-Disorders/Pompe-Disease-Information-Page. Accessed May 2019. THIS IS JUST THE BEGINNING OF OUR COMMITMENT

40 TOMORROW’S PROMISE: 40 PROGRAMS THAT COULD IMPACT MILLIONS OF PATIENTS AROUND THE GLOBE

GOAL EPIDEMIOLOGY* 40 >1.5M patients with Programs rare disease globally

*Based on published epidemiology

13 LOCATIONS EMPLOYEES >600 (and growing!)

43 “We choose to go to the moon. We choose to go to the moon in this decade and do the other things, not because they are easy, but because they are hard, because that goal will serve to organize and measure the best of our energies and skills, because that challenge is one that we are willing to accept, one we are unwilling to postpone, and one which we intend to win...”

PRESIDENT JOHN F. KENNEDY Address at Rice University September 12, 1962

45 DRAGGING TOMORROW INTO TODAY

#DraggingTomorrowIntoToday