Genetic Disorders
eliglustat (Cerdelga®) | ||
---|---|---|
eliglustat (Cerdelga®) Approval Criteria:
|
imiglucerase (Cerezyme®), taliglucerase alfa (Elelyso®), and velaglucerase alfa (Vpriv®) | ||
---|---|---|
imiglucerase (Cerezyme®), taliglucerase alfa (Elelyso®), and velaglucerase alfa (Vpriv®) Approval Criteria:
|
miglustat (Zavesca®) | ||
---|---|---|
miglustat (Zavesca®) Approval Criteria:
|
eteplirsen (Exondys 51™) | ||
---|---|---|
eteplirsen (Exondys 51™) Approval Criteria:
|
deflazacort (Emflaza®) | ||
---|---|---|
deflazacort (Emflaza®) Approval Criteria:
|
tolvaptan (Jynarque™) | ||
---|---|---|
Jynarque™ (Tolvaptan) Approval Criteria:
|
nusinersen (Spinraza™) | ||
---|---|---|
nusinersen (Spinraza™) Approval Criteria:
|
burosuman-twza (Crysvita®) | ||
---|---|---|
Crysvita® (Burosumab-twza) Approval Criteria:
|
Alpha1-Proteinase Inhibitor [Human] | ||
---|---|---|
Prolastin®-C (Alpha1-Proteinase Inhibitor [Human]) Approval Criteria:
Prolastin®-C Liquid [Alpha1-Proteinase Inhibitor (Human)] Approval Criteria:
Aralast NP™,Glassia®, and Zemaira® (Alpha1-Proteinase Inhibitor [Human]) Approval Criteria:
|
l-glutamine (Endari™) | ||
---|---|---|
Approval Criteria:
|
l-glutamine (NutreStore®) | ||
---|---|---|
NutreStore® (L-Glutamine) Approval Criteria [Short Bowel Syndrome Diagnosis]:
NutreStore® (L-Glutamine) Approval Criteria [Sickle Cell Disease Diagnosis]:
|
hydroxyurea tablets (Siklos®) | ||
---|---|---|
hydroxyurea tablets(Siklos®) Approval Criteria:
|
eculizumab (Soliris®) | ||
---|---|---|
eculizumab (Soliris®) Approval Criteria [Generalized Myasthenia Gravis (gMG) Diagnosis]:
|
voretigene neparvovec-rzyl (Luxturna™) | ||
---|---|---|
Luxturna™ (Voretigene Neparvovec-rzyl) Approval Criteria:
|