Orphan Disease and Orphan Drugs Flashcards
1
Q
CAUSE
Gaucher’s Disease
A
Glucocebrosidase
2
Q
CAUSE
Fabry’s DiseasE
A
Galactosidase
3
Q
CAUSE
Mucopolysaccharidosis
A
Lysosomal enzymes
4
Q
CAUSE
Tourette’s Syndrome
A
Motor tics
5
Q
CAUSE
Crohn’s Syndrome
A
unknown
6
Q
CAUSE
Wilson’s Disease
A
Copper deposition
7
Q
CAUSE
SCID
A
Adenosine Deaminase
8
Q
ORPHAN DRUG
Gaucher’s Disease
A
miglustat
9
Q
ORPHAN DRUG
Fabry’s Disease
A
Galsidase β
10
Q
ORPHAN DRUG
Mucopolysaccharidosis
A
Laronidase
11
Q
ORPHAN DRUG
Tourette’s Syndrome
A
Lamotrizine
12
Q
ORPHAN DRUG
Crohn’s Syndrome
A
Infliximab
13
Q
ORPHAN DRUG
Wilson’s Disease
A
Trientine
14
Q
A
