Enter Title

Dosing, Administration & Timing

100

formulation & route of administration of Migalastat

Oral Capsules

200

how often Migalastat should be taken by patients with Fabry diseases

Every other day

300

food consumption have that effect on the pharmacokinetics of Migalastat

Reduced absorption

400

Galafold SmPC says that about food consumption

Take Migalastat at least 2hrs before or after food

MOA, Indications & Contraindications

100

Replagal is ERT, but Migalastat is that

Chaperone

200

it is the key genetic feature of Fabry disease patients who are candidates for Migalastat

having an amenable mutation

300

which of the following is NOT a feature of MOA of Migalastat?

-decrease alpha-Gal A activity

-stabilizes mutant alpha-Gal A

-promotes folding, stabilization & lysosomal trafficking of mutant forms of alpha-Gal A

-act as selective, reversible competitive inhibitor of alpha-Gal A

-decrease alpha-Gal A activity

400

limitations to prescribing Migalastat

-Age<65 years & eGFR>60 mL/min/1.73m²

-Age<16 years & eGFR<60 mL/min/1.73m²

-Age<16 years & eGFR<30 mL/min/1.73m²

-Age<65 years & eGFR>30 mL/min/1.73m²

-Age<16 years & eGFR<30 mL/min/1.73m²

Migalastat Vs. Replagal

100

the period of follow-up that Replagal has demonstrated benefits in Cardiac structure, symptoms & function

10 years

200

Replagal demonstrated which of the following

-improved cerebral blood flow

-improvements in cardiac function & rhythm

-reduced risk of events, including strokes

-all of the above

-all of the above

300

the only drug class that was studied with Migalastat from a drug-drug interaction prespective

ERT

400

True or False

no evidence so far that it is necessary for a therapeutic agent to cross BBB to reduce the risk of stroke in Fabry disease

True

FACETS

100

patients included in FACETS had to be ERT-naive or ERT-free for how long before entering the study

at least 6 months

200

the impact of Migalastat on renal function in FACETS & longer term extension studies

renal functions remained stable

300

renal outcomes measured as a secondary endpoints during FACETS

eGFR, mGFR & 24 hrs urine protein

400

proportion of patients treated with Migalastat in FACETS who achieved >50% reduction from baseline in kidney interstitial capillary Gb3(primary endpoint) in the initial ITT population analysis

41%