Table 2 Results of the questionnaire assessing the Polish experts’ opinion regarding migalastat use in Fabry disease (FD); the level of agreement is presented as percentage of physicians (n = 5) prescribing migalastat

When compared with i.v., oral therapy can improve QoL in patients with FD

According to available data, migalastat can be considered a safe and effective treatment for FD

According to available evidence, one of the advantages of migalastat over ERT is its superior efficacy on heart damage

Poor compliance to oral therapy with migalastat can be an issue

Migalastat therapy can be taken into consideration as an alternative to ERT in patients with FD and amenable mutations

In a male patient aged ≥ 12 years with an amenable mutation and classic FD, migalastat therapy might be taken into consideration at diagnosis, even when signs/symptoms of organ damage are lacking

In a male or female patient aged ≥ 12 years with an amenable mutation and classic FD, migalastat therapy is recommended in the presence of signs/symptoms of organ damage

In a male patient aged ≥ 12 years with an amenable mutation and non-classic FD, migalastat therapy is recommended in the presence of signs/symptoms of organ damage

In a female patient aged ≥ 12 years with an amenable mutation and non-classic FD, migalastat therapy might be taken into consideration at diagnosis, even when signs/symptoms of organ damage are lacking

In a female patient aged ≥ 12 years with an amenable mutation and non-classic FD, migalastat therapy might be considered at the first onset of signs/symptoms of organ damage

Migalastat therapy is recommended in a patient with FD aged ≥ 12 years, with an amenable mutation and cardiac hypertrophy (≥ 11 mm)

Migalastat treatment should be considerated in a patient aged ≥ 12 years with FD, with an amenable mutation and rhythm disorders (sinus bradycardia, atrial fibrillation, extrasystole) and/or electrocardiogram alterations

Migalastat therapy is recommended in a patient with FD aged ≥ 12 years with an amenable mutation and pathological microalbuminuria (according to KDIGO guidelines)

Migalastat therapy is recommended in a patient with FD aged ≥ 12 years with an amenable mutation and proteinuria (according to KDIGO guidelines)

Migalastat therapy is recommended in patients aged ≥ 12 years with FD, amenable mutations and eGFR 60–90 ml/min/1.73 m² (CKD-EPI) with evidence of renal function decline progression (> − 1 ml/min/1.73 m²/year)

Migalastat therapy is recommended in patients aged ≥ 12 years with FD, amenable mutations and eGFR 30–60 ml/min/1.73 m² (CKD-EPI)

Migalastat treatment may be considered in patients aged ≥ 12 years with FD, amenable mutations and progression of white matter lesions

Migalastat treatment may be considered in patients aged ≥ 12 years with FD, amenable mutations and history of TIA/stroke

Migalastat treatment may be considered in patients aged ≥ 12 years with FD, amenable mutations and progressive loss of hearing (corrected by age)

Migalastat treatment should be considered in patients aged ≥ 12 years with FD, amenable mutations and gastrointestinal symptoms

Migalastat treatment should be considerated in patients aged ≥ 12 years with FD, amenable mutations and acroparesthesia, even if controlled by symptomatic therapy

In a patient aged ≥ 12 years with an amenable mutation, currently on ERT, switching to migalastat should be considered in unstable disease and/or poor therapy response

In a patient aged ≥ 12 years with an amenable mutation, currently on ERT, switching to migalastat should be considered in uncontrolled infusion reactions and/or poor compliance

In a patient aged ≥ 12 years with an amenable mutation, currently on ERT, switching to migalastat should be considered in movement/mobility impairment affecting difficulties in transport for ERT infusions