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Hunter Syndrome: Why We Need to Diagnose and Treat Early

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Presentation on theme: "Hunter Syndrome: Why We Need to Diagnose and Treat Early"— Presentation transcript:

1 Hunter Syndrome: Why We Need to Diagnose and Treat Early

2

3 Hunter Syndrome (MPS II)

4 Hunter Syndrome: Clinical Presentation

5 Hunter Syndrome: Major Signs and Symptoms

6 Severe Hunter Syndrome

7 Challenges of Early Diagnosis

8 Delay in Recognizing the Diagnosis

9 'Red Flag' Signs and Symptoms of Hunter Syndrome That Occur Early in Disease Course

10 ENT and Hernia Surgeries in Children with Hunter Syndrome: Data from Hunter Outcome Survey

11 HUNTER Mnemonic Screening Instrument

12 Suspecting Hunter Syndrome to Establish an Early Diagnosis

13 Family History of Lysosomal Storage Disease

14 Hunter Syndrome: Diagnosis and Work-Up

15 Hunter Syndrome: Laboratory Diagnostic Testing

16 Diagnostic Challenge: Global Availability of Genetic Testing

17 Glycosaminoglycan Assays for MPS

18 Newborn Screening of MPS

19 Early Treatment of Hunter Syndrome[a]

20 Hunter Syndrome: Treatments

21 Challenges Related to ERT

22 Initiate ERT as Early as Possible

23 Early Treatment With ERT (<5 Years of Age): Growing Clinical Experience

24 Early Treatment With ERT (<5 Years of Age): Safety Experience

25 Severe Hunter Syndrome: CNS Involvement

26 Hunter Syndrome: CNS Symptom-Based Interventions

27 Summary: Diagnosis and Existing Treatment

28 Therapeutic prospects for patients with Hunter Syndrome

29 AIM-IT: IT Idursulfase

30 AIM-IT: Preliminary Data

31 Improving Outcomes for Patients With Hunter Syndrome

32 Multidisciplinary Management of Patients With Hunter Syndrome

33 Abbreviations


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