Israel Alfonso, MD INSTRUCTIONS READ EACH QUESTION

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Presentation transcript:

Israel Alfonso, MD INSTRUCTIONS READ EACH QUESTION GIVE IT YOUR BEST SHOT FOLLOW THE ARROWS GOOD LUCK! Israel Alfonso, MD

1. MUCOPOLYSACCHARIDES, ALSO KNOWN AS ______________ ARE THE BY PRODUCT OF COLLAGEN CATABOLISM: A. MUCOLIPIDS B. SPHINGOLIPIDS C. OLIGOSACCHARIDES D. GLYCOSAMINOGLYCANS

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COLLAGEN FIBER HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE PROTEOLYTIC ENZYMES COLLAGEN FIBER HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE THIS SUBSTANCES ARE CALLED MUCOPOLYSACCHARIDES OR GLYCOSAMINOGLYCANS

1. MUCOPOLYSACCHARIDES ALSO KNOWN AS ______________ ARE THE BY PRODUCT OF COLLAGEN CATABOLISM: A. MUCOLIPIDS B. SPHINGOLIPIDS C. OLIGOSACCHARIDES D. GLYCOSAMINOGLYCANS

2. WHICH OF THE FOLLOWING CONDITIONS IS X-LINKED: A. HURLER SYNDROME B. SCHEIE SYNDROME C. HUNTER SYNDROME D. MORQUIO SYNDROME

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X-LINKED ONLY BOYS HUNTER SYNDROME BOYS ARE GOOD HUNTERS THE ONLY OTHER X-LINKED LYSOSOMAL DISORDER IS FABRY

2. WHICH OF THE FOLLOWING CONDITIONS IS X-LINKED: A. HURLER SYNDROME B. SCHEIE SYNDROME C. HUNTER SYNDROME D. MORQUIO SYNDROME

3. A DEFICIENCY OF a -L- IDURONIDASE PRODUCES : A. HURLER SYNDROME B. HUNTER SYNDROME C. SANFILIPPO B D. SANFILIPPO A

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A-L-IDURONIDASE HURLER COARSE FACE DYSOSTOSIS MULTIPLEX LIVER BIG

3. A DEFICIENCY OF a -L- IDURONIDASE PRODUCES : A. HURLER SYNDROME B. HUNTER SYNDROME C. SANFILIPPO B D. SANFILIPPO A

4. WHICH OF THE FOLLOWING IS NOT ASSOCIATED WITH HURLER SYNDROME? A. BRACHIOCEPHALY B. HYPERTELORISM C. HEARING LOSS D. CORNEA CLOUDING

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FACIAL FEATURES OF HURLER SYNDROME CORNEAL CLOUDING FLAT NOSE BRIDGE LARGE NARES THICK LIPS APPEARS FROM 6 TO 24 MONTHS DEAFNESS SCAPHOCEPHALY HEAVY EYE BROWS HYPERTELORISM PROMINENT TONGUE PROMINENT SUPRAORBITAL RIDGES

FACIAL FEATURES OF HUNTER SYNDROME FLAT NOSE BRIDGE LARGE NARES THICK LIPS LESS PROMINENT APPEARS FROM 2 TO 4 YEARS SCAPHOCEPHALY HEAVY EYE BROWS HYPERTELORISM PROMINENT TONGUE PROMINENT SUPRAORBITAL RIDGES

4. WHICH OF THE FOLLOWING IS NOT ASSOCIATED WITH HURLER SYNDROME? A. BRACHIOCEPHALY B. HYPERTELORISM C. HEARING LOSS D. CORNEA CLOUDING

5. WHICH OF THE FOLLOWING IS MORE COMMON IN INFANTS WITH HURLER SYNDROME: A. CARDIOMYOPATHY B. LIVER FAILURE C. FREQUENT BONE FRACTURES D. ASTHMA

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DUE TO DEPOSITION OF DERMATAN AND HEPARAN SULFATE IN THE MYOCARDIUM AND VALVES

5. WHICH OF THE FOLLOWING IS MORE COMMON IN INFANTS WITH HURLER SYNDROME: A. CARDIOMYOPATHY B. LIVER FAILURE C. FREQUENT BONE FRACTURES D. ASTHMA

6. CHILDREN WITH HURLER SYNDROME FREQUENTLY HAVE UPPER RESPIRATORY SYMPTOMS AND OTITIS MEDIA, WHY? A. BAD IMMUNOLOGICAL SYSTEM B. ABNORMAL BONY CALVARIUM C. POOR HYGIENE D. ALLERGIES

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CRANIAL DYSOSTOSIS SAGITTAL SYNOSTOSIS ENLARGE J-SHAPED SELLA THICK HYPOPLASIA OF MIDFACIAL BONES PARTIAL LAMBDOIDAL SYNOSTOSIS

6. CHILDREN WITH HURLER SYNDROME FREQUENTLY HAVE UPPER RESPIRATORY SYMPTOMS AND OTITIS MEDIA, WHY? A. BAD IMMUNOLOGICAL SYSTEM B. ABNORMAL BONY CALVARIUM C. POOR HYGIENE D. ALLERGIES

7. DYSTOSIS MULTIPLEX IN PATIENTS WITH HURLER SYNDROME IS CHARACTERIZED BY ALL OF THE FOLLOWING EXCEPT: A. OAR SHAPED RIBS B. ANTERIOR HYPOPLASIA OF THE LUMBAR VERTEBRAS C. HYPOPLASTIC ILIA D. LARGE FEMORAL HEADS

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HURLER SYNDROME LONG BONES: BROAD AND SHORT ANTERIOR HYPOPLASIA OF THE VERTEBRAS AND KYPHOSIS BROAD, BULLET LIKE PHALANGES HYPOBLASTIC ILIA & FEMORAL HEAD

HURLER SYNDROME OAR-SHAPED RIBS ARE NARROW AT THE VERTEBRAL END AND BROAD AT THE STERNAL AGE

7. DYSTOSIS MULTIPLEX IN PATIENTS WITH HURLER SYNDROME IS CHARACTERIZED BY ALL OF THE FOLLOWING EXCEPT: A. OAR SHAPED RIBS B. ANTERIOR HYPOPLASIA OF THE LUMBAR VERTEBRAS C. HYPOPLASTIC ILIA D. LARGE FEMORAL HEADS

8. WHICH OF THE FOLLOWING SUBSTANCE IS EXCRETED IN THE URINE OF CHILDREN WITH HURLER SYNDROME? A. HEPARAN AND KERATAN B. KERATAN AND CHONDROTIN C. DERMATAN AND HEPARAN D. HEPARAN AND CHONDROTIN

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4p16.3 ENZYMES GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER HURLER PROTEOLYTIC ENZYMES COLLAGEN FIBER HEPARAN SULFATE DERMATAN SULFATE a-L-IDURONIDASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE HURLER SCHEIE HURLER-SCHEIE 4p16.3 SIMPLE SUGAR

8. WHICH OF THE FOLLOWING SUBSTANCE IS EXCRETED IN THE URINE OF CHILDREN WITH HURLER SYNDROME? A. HEPARAN AND KERATAN B. KERATAN AND CHONDROTIN C. DERMATAN AND HEPARAN D. HEPARAN AND CHONDROTIN

9. THE GENE ENCODING a –L-IDURONIDASE, THE ENZYME INVOLVED IN HURLER SYNDROME IS LOCATED AT : A. 4p16.3 B. Xq28 C. 17q25.3 D. 7q21.1

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4p16.3 ENZYMES GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER HURLER PROTEOLYTIC ENZYMES COLLAGEN FIBER HEPARAN SULFATE DERMATAN SULFATE a-L-IDURONIDASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE HURLER SCHEIE HURLER-SCHEIE 4p16.3 SIMPLE SUGAR

4p16.3 THE ONLY ONE OF THE MUCOPOLYSACCHARIDOSIS WITH GENE LOCATION IN THE SMALL ARM (p).

9. THE GENE ENCODING a –L-IDURONIDASE, THE ENZYME INVOLVED IN HURLER SYNDROME IS LOCATED AT : A. 4p16.3 B. Xq28 C. 17q25.3 D. 7q21.1

10. THE MOST FREQUENT CAUSE OF HYDROCEPHALUS IN PATIENTS WITH HURLER SYNDROME IS? A. AQUEDUCTAL STENOSIS B. OBSTRUCTION OF THE ORIFICE OF MONROE C. NON-COMMUNICATING HYDROCEPHALUS D. COMMUNICATING HYDROCEPHALUS

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GOOD CSF FLOW (WHITE) TO THE CISTERNA MAGNA LATERAL VENTRICLE AQUEDUCT OF SILVIUS ORIFICE OF MONROE 3rd VENTRICLE 4th VENTRICLE CISTERNA MAGNA COMMUNICATING HYDROCEPHALUS

HURLER SYNDROME STIFF JOINTS HERNIA

NEUROLOGICAL DETERIORATION IN A PATIENT WITH HURLER SYNDROME MAY HAVE TWO CAUSES: 1) DEPOSITION OF GANGLIOSIDES IN THE NEURON (MECHANISM UNKNOWN, NATURAL COURSE OF THE DISEASE), 2) DEPOSITION OF MUCOPOLYSACCHARIDES IN THE MENINGES THUS BLOCKING THE INLETS OF THE CORPUSCLE OF PACCHIONI, OR 3) A COMBINATION

10. THE MOST FREQUENT CAUSE OF HYDROCEPHALUS IN PATIENTS WITH HURLER SYNDROME IS? A. AQUEDUCTAL STENOSIS B. OBSTRUCTION OF THE ORIFICE OF MONROE C. NON-COMMUNICATING HYDROCEPHALUS D. COMMUNICATING HYDROCEPHALUS

11. CORNEAL CLOUDING IS NOT A FEATURE OF? A. HURLER SYNDROME B. HURLER-SCHEIE SYNDROME C. SCHEIE SYNDROME D. HUNTER SYNDROME

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OSTEO DYSTOSIS JOINT STIFFNESS MYOCARDIOPATHY HEPATOMEGALY MORQUIO SYNDROME SCHEIE SYNDROME HURLER-SCHEIE SYNDROME HURLER SYNDROME OSTEO DYSTOSIS JOINT STIFFNESS MYOCARDIOPATHY HEPATOMEGALY MENTAL RETARDATION CORNEAL CLOUDINESS

11. CORNEAL CLOUDING IS NOT A FEATURE OF? A. HURLER SYNDROME B. HURLER-SCHEIE SYNDROME C. SCHEIE SYNDROME D. HUNTER SYNDROME

12. WHICH OF THE FOLLOWING SUBSTANCE IS EXCRETED IN THE URINE OF CHILDREN WITH HUNTER SYNDROME? A. HEPARAN AND KERATAN B. KERATAN AND CHONDROTIN C. DERMATAN AND HEPARAN D. HEPARAN AND CHONDROTIN

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X Xq28 GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER HUNTER PROTEOLYTIC ENZYMES COLLAGEN FIBER HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE IDURONATE SULFATASE HEPARAN SULFATE DERMATAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE HUNTER Xq28 X

FACIAL FEATURES OF HUNTER SYNDROME LESS PROMINENT THAN HURLER SCAPHOCEPHALY LARGE NARES NO CORNEAL CLOUDING THICK LIPS APPEARS FROM 2 TO 4 YEARS FLAT NOSE BRIDGE COMMUNICATING HYDROCEPHALUS HEAVY EYE BROWS HYPERTELORISM PROMINENT TONGUE PROMINENT SUPRAORBITAL RIDGES

12. WHICH OF THE FOLLOWING SUBSTANCE IS EXCRETED IN THE URINE OF CHILDREN WITH HUNTER SYNDROME? A. HEPARAN AND KERATAN B. KERATAN AND CHONDROTIN C. DERMATAN AND HEPARAN D. HEPARAN AND CHONDROTIN

13. SHE IS A 6 YEAR OLD GIRL WITH HUNTER SYNDROME. A. TRUE B. FALSE

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FACIAL FEATURES OF HUNTER SYNDROME LESS PROMINENT THAN HURLER SCAPHOCEPHALY LARGE NARES NO CORNEAL CLOUDING THICK LIPS APPEARS FROM 2 TO 4 YEARS FLAT NOSE BRIDGE COMMUNICATING HYDROCEPHALUS HEAVY EYE BROWS HYPERTELORISM PROMINENT TONGUE PROMINENT SUPRAORBITAL RIDGES

HOW TO REMEMBER GIRLS DO NOT LIKE TO HUNT, SO THEY ARE NOT HUNTERS (ONLY BOYS). HUNTERS NEED GOOD VISION, SO THEY DO NOT HAVE CORNEAL CLOUDING.

13. SHE IS A 6 YEAR OLD GIRL WITH HUNTER SYNDROME. A. TRUE B. FALSE

14. THE ONSET OF FINDINGS OF HUNTER SYNDROME IS USUALLY BY? A. 2 TO 4 YEARS OF AGE B. 6 AND 24 MONTHS OF AGE C. FIRST 6 MONTHS OF LIFE D. 2 TO 6 YEARS OF AGE

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AGE OF ONSET SANFILIPPO HUNTER HURLER GM1 GANGLIOSIDOSIS 1 2 3 4 5 6 YEARS

14. THE ONSET OF FINDINGS OF HUNTER SYNDROME IS USUALLY BY? A. 2 TO 4 YEARS OF AGE B. 6 AND 24 MONTHS OF AGE C. FIRST 6 MONTHS OF LIFE D. 2 TO 6 YEARS OF AGE

15. MILD HEPATOMEGALY, HIRSUTISM OR BOTH IN A 6-YEAR-OLD WITH HYPERACTIVITY SHOULD MAKE YOU THINK OF? A. SCHEIE SYNDROME B. HURLER SYNDROME C. HUNTER SYNDROME D. SANFILIPPO SYNDROME

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SCHEIE NORMAL MENTAL DELAY + HURLER MENTAL DELAY +/- HUNTER SAN- FILIPPO HYPER/ AGGRESSIVE

15. MILD HEPATOMEGALY, HIRSUTISM OR BOTH IN A 6-YEAR-OLD WITH HYPERACTIVITY SHOULD MAKE YOU THINK OF? A. SCHEIE SYNDROME B. HURLER SYNDROME C. HUNTER SYNDROME D. SANFILIPPO SYNDROME

16. WHICH OF THE FOLLOWING GLYCOSAMINOGLYCAN IS STORED IN SANFILIPPO SYNDROME? A. DERMATAN AND HEPARAN B. DERMATAN C. HEPARAN D. KERATAN AND DERMATAN

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17q25.3 GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER SANFILIPPO A PROTEOLYTIC ENZYMES COLLAGEN FIBER HEPARAN N-SULFATASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE 17q25.3 SANFILIPPO A

17q21. GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER SANFILIPPO B PROTEOLYTIC ENZYMES COLLAGEN FIBER a-N-ACYL GLUCOSAMINIDASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE 17q21. SANFILIPPO B

GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER PROTEOLYTIC ENZYMES COLLAGEN FIBER ACETYL-COa ACYL TRANSFERASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE SANFILIPPO C

GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER PROTEOLYTIC ENZYMES COLLAGEN FIBER N-ACYL GLUCOSAMINE 6- SULFATASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE SANFILIPPO D

16. WHICH OF THE FOLLOWING GLYCOSAMINOGLYCAN IS STORED IN SANFILIPPO? A. DERMATAN AND HEPARAN B. DERMATAN C. HEPARAN D. KERATAN AND DERMATAN

17. ALL OF THE FOLLOWING ENZYMES MAY BE INVOLVED SANFILIPPO SYNDROME, EXCEPT? A. HEPARAN-N-SULFATE B. a-N-ACETYL-GLUCOSAMINIDASE C. N-ACETYLGLUCOSAMINE 6 SULFATASE D. b-GALACTOSIDASE

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17q25.3 GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER SANFILIPPO A PROTEOLYTIC ENZYMES COLLAGEN FIBER HEPARAN N-SULFATASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE 17q25.3 SANFILIPPO A

17q21. GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER SANFILIPPO B PROTEOLYTIC ENZYMES COLLAGEN FIBER a-N-ACYL GLUCOSAMINIDASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE 17q21. SANFILIPPO B

GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER PROTEOLYTIC ENZYMES COLLAGEN FIBER ACETYL-COa ACYL TRANSFERASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE SANFILIPPO C

12q14. GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER SANFILIPPO D PROTEOLYTIC ENZYMES COLLAGEN FIBER N-ACYL GLUCOSAMINE 6- SULFATASE HEPARAN SULFATE DERMATAN SULFATE HEPARAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE 12q14. SANFILIPPO D

17. ALL OF THE FOLLOWING ENZYMES MAY BE INVOLVED SANFILIPPO SYNDROME, EXCEPT? A. HEPARAN-N-SULFATE B. a-N-ACETYL-GLUCOSAMINIDASE C. N-ACETYLGLUCOSAMINE 6 SULFATASE D. b-GALACTOSIDASE

18. WHICH OF THE FOLLOWING IS NOT A FEATURE OF MORQUIO SYNDROME? A. CORNEAL CLOUDING B. MENTAL RETARDATION C. SHORT-TRUNK DWARFISM D. SKELETAL DYSPLASIA

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MORQUIO CORNEAL CLOUDING

MORQUIO

MORQUIO

18. WHICH OF THE FOLLOWING IS NOT A FEATURE OF MORQUIO SYNDROME? A. CORNEAL CLOUDING B. MENTAL RETARDATION C. SHORT-TRUNK DWARFISM D. SKELETAL DYSPLASIA

19. WHICH OF THE FOLLOWING IS THE MOST PROMINENT FEATURE OF SANFILIPPO SYNDROME? A. CORNEAL CLOUDING B. MENTAL RETARDATION C. SHORT-TRUNK DWARFISM D. SKELETAL DYSPLASIA

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SANFILIPPO SEVERE CNS INVOLVEMENT WITH LITTLE SYSTEMIC INVOLVEMENT 6/15/97

SANFILIPPO 6/15/89 6/15/97 MINIMAL BONE INVOLVEMENT

19. WHICH OF THE FOLLOWING IS THE MOST PROMINENT FEATURE OF SANFILIPPO SYNDROME? A. CORNEAL CLOUDING B. MENTAL RETARDATION C. SHORT-TRUNK DWARFISM D. SKELETAL DYSPLASIA

20. THE GENE FOR SANFILIPPO SYNDROME A AND B ARE AT CHROMOSOME?

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SANFILIPPO A 17q25.3 SEVERE CNS INVOLVEMENT WITH LITTLE SYSTEMIC INVOLVEMENT 6/15/97

MORQUIO 16q24.3 NO MENTAL RETARDATION

20. THE GENE FOR SANFILIPPO SYNDROME A AND B ARE AT CHROMOSOME?

21. ALL OF THE FOLLOWING ARE EXTRASKELETAL MANIFESTATION OF MORQUIO SYNDROME, EXCEPT? A. SPLENOMEGALY B. CORNEAL CLOUDING C. HEPATOMEGALY D. SMALL THIN TEETH

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CORNEAL CLOUDING SMALL THIN TEETH HEPATOMEGALY CARDIOVASCULAR LESION FREQUENT CARIES

21. ALL OF THE FOLLOWING ARE EXTRASKELETAL MANIFESTATION OF MORQUIO SYNDROME, EXCEPT? A. SPLENOMEGALY B. CORNEAL CLOUDING C. HEPATOMEGALY D. SMALL THIN TEETH

22. WHICH OF THE FOLLOWING IS THE MOST FREQUENT MECHANISM OF CORD COMPRESSION IN MAROTEAUX-LAMY SYNDROME? A. THICKENING OF THE DURA B. C1-C2 SUBLUXATION C. SPINAL CORD STROKE D. SPINAL CORD BLEEDING

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MORQUIO 16q24.3 C1-C2 SUBLUXATION NO MENTAL RETARDATION

MAROTEAUX-LAMY SYNDROME HURLER-LIKE THAT LAGS IN DEVELOPING MENTAL RETARDATION (5 q13-14) CORD COMPRESSION DUE TO DURAL THICKENING ODONTOID HYPOPLASIA ALSO OCCURS

22. WHICH OF THE FOLLOWING IS THE MOST FREQUENT MECHANISM OF CORD COMPRESSION IN MAROTEAUX-LAMY SYNDROME? A. THICKENING OF THE DURA B. C1-C2 SUBLUXATION C. SPINAL CORD STROKE D. SPINAL CORD BLEEDING

23. WHICH OF THE FOLLOWING LYSOSOMAL STORAGE DISEASES MAY BE PRESENT AT BIRTH? A. HURLER SYNDROME B. HUNTER SYNDROME C. MORQUIO SYNDROME D. SLY SYNDROME

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URINE OLIGOSACCHARIDES HYDROPS FETALIS URINE OLIGOSACCHARIDES YES SIALIDOSIS II NO GALACTOSIALIDOSIS a- MANNOSIDOSIS SERUM OR URINE FREE SIALIC ACIDS SIALIC ACID STORAGE DISEASE SERUM HIGH ACID PHOSPHATASE GAUCHER DISEASE SERUM LYSOSOMES ENZYMES I-CELL DISEASE HIGH DERMATAN & HEPARAN SULFATE IN URINE & METACHROMATIC INCLUSION IN GRANULOCYTES SLY DISEASE Gilles L, Adams RD, Kolodny EH. Neurology of Hereditary Metabolic Diseases of the Child. McGraw-Hill, NY 1996

SLY SYNDROME THEY LOOK LIKE PATIENTS WITH HURLERS SYNDROME BUT FROM BIRTH OR EVEN PRENATALLY, THEY USUALLY HAVE HYDROPS FETALIS 7q21.1

23. WHICH OF THE FOLLOWING LYSOSOMAL STORAGE DISEASES MAY BE PRESENT AT BIRTH? A. HURLER SYNDROME B. HUNTER SYNDROME C. MORQUIO SYNDROME D. SLY SYNDROME

24. WHICH OF THE FOLLOWING ENZYMES IS DEFICIENT IN SLY SYNDROME? A. a-L-IDURONIDASE B. b-GLUCURONIDASE C. b-GALACTOSIDASE D. GALACTOSE-6-SULFATASE

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7q21.1 GLYCOSAMINOGLYCANS CATABOLISM COLLAGEN FIBER SLY PROTEOLYTIC ENZYMES COLLAGEN FIBER DERMATAN SULFATE CHONDROTIN SULFATE HEPARAN SULFATE HEPARAN SULFATE b GLUCURONIDASE HEPARAN SULFATE DERMATAN SULFATE DERMATAN SULFATE KERATAN SULFATE CHONDROTIN SULFATE 7q21.1 SLY

24. WHICH OF THE FOLLOWING ENZYMES IS DEFICIENT IN SLY SYNDROME? A. a-L-IDURONIDASE B. b-GLUCURONIDASE C. b-GALACTOSIDASE D. GALACTOSE-6-SULFATASE

25. WHICH OF THE FOLLOWING SUBSTANCES IS STORED IN THE CENTRAL NERVOUS SYSTEM LYSOSOMES OF PATIENTS WITH MUCOPOLYSACCHARIDOSIS? A. GANGLIOSIDE B. HEPARAN C. DERMATAN D. KERATAN

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GANGLIOSIDE

HEPARAN, DERMATAN AND KERATAN DO NOT CROSS THE BLOOD BRAIN BARRIER

HEPARAN, DERMATAN AND KERATAN MAY ACCUMULATE IN THE MENINGEAL CELLS AND AS YOU KNOW PRODUCE HYDROCEPHALUS

25. WHICH OF THE FOLLOWING SUBSTANCES IS STORED IN THE CENTRAL NERVOUS SYSTEM LYSOSOMES OF PATIENTS WITH MUCOPOLYSACCHARIDOSIS? A. GANGLIOSIDE B. HEPARAN C. DERMATAN D. KERATAN

26. PRENATAL DIAGNOSIS OF ALL MUCOPOLYSACCHARIDOSIS IS POSSIBLE? A. TRUE B. FALSE

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FROM CULTURED AMNIOTIC CELLS CHRONIC VILLUS BIOPSY MEASUREMENT OF GLYCOAMINOGLYCANS

26. PRENATAL DIAGNOSIS OF ALL MUCOPOLYSACCHARIDOSIS IS POSSIBLE? A. TRUE B. FALSE

27. THE INDICATION OF BONE MARROW TRANSPLANTATION INCLUDE: A. BONE PROBLEMS B. EYE PROBLEMS C. HURLER SYNDROME, LESS THAN 24 MONTHS AND MENTALLY FINE D. HUNTER SYNDROME

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ENZYME THERAPY IS COMINGS HURLER SYNDROME ENZYME THERAPY IS COMINGS

27. THE INDICATIONS OF BONE MARROW TRANSPLANTATION INCLUDE: A. BONE PROBLEMS B. EYE PROBLEMS C. HURLER SYNDROME, LESS THAN 24 MONTHS AND MENTALLY FINE D. HUNTER SYNDROME

THERE ARE NO MORE QUESTIONS THE END I HOPE YOU HAVE LEARNED ALL YOU NEED TO KNOW ABOUT MUCOPOLYSACCHARIDOSIS