natgillin/translations-raw huggingface dataset:
Table 2: Galafold (migalastat) amenability table 
Tabuľka 2: Tabuľka senzibility lieku Galafold (migalastát)
Do not take Galafold on two consecutive days. 
Galafold dva po sebe nasledujúce dni.
c.924A>T or c.924A>C 
c.924A>T alepo c.924A>C
c.611G>A or 612G>A 
c.611G>A alepo 612G>A
c.A124C or c.A124T 
c.A124C alepo c.A124T
c.369+3_639+954del3129insT UNKNOWN (del Exon3 and 4?) 
c.369+3_639+954del3129insT
After follow up in the OLE, the mean change from baseline in LVMi at Month 36 was -8.3 (95% CI: -17.1, 0.4; n=25) and at Month 48 was -9.1 (95% CI: -20.3, 2.0; n=18). 
Po sledovaní v OLE bola priemerná zmena LVMi od začiatku do 36. mesiaca -8,3 (95 % IS: -17,1; 0,4; n=25) a do 48. mesiaca -9,1 (95%
c.C525G or c.C525A 
c.C525G alepo c.C525A
c.1147T>C or c.1149C>G or c.1149C>A 
c.1147T>C alepo c.1149C>G alepo c.1149C>A
c.T187A or c.G188C 
c.T187A alepo c.G188C
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c.G261C or c.G261T 
c.G261C alepo c.G261T
It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on or switched to Galafold. 
U pacientov, v prípade ktorých sa začala liečba liekom Galafold alebo ktorí prešli na liečbu liekom Galafold, sa odporúča pravidelne sledovať funkciu obličiek, echokardiografické parametre a biochemické markery (každých šesť mesiacov).
What you need to know before you take Galafold 3. 
1. Čo je Galafold a na čo sa používa 2. Čo potrebujete vedieť predtým, ako užijete Galafold 3. Ako užívať Galafold
c.G982A or c.G982C 
c.G982A alepo c.G982C
c.C305G or c.C305A 
c.C305G alepo c.C305A
c.187T>A or c.188G>C 
c.187T>A alepo c.188G>C
c.G285T or c.G285C 
c.G285T alepo c.G285C
c.285G>T or c.285G>C 
c.285G>T alepo c.285G>C
c.216G>A or c.216G>T or c.216G>C 
c.216G>A alepo c.216G>T alepo c.216G>C
c.T166A or c.G167C 
c.T166A alepo c.G167C
c.261G>C or c.261G>T 
c.261G>C alepo c.261G>T
c.G540C or c.G540T 
c.G540C alepo c.G540T
c.280T>A or c.281G>C 
c.280T>A alepo c.281G>C
c.305C>G or c.305C>A 
c.305C>G alepo c.305C>A
c.G132T or c.G132C 
c.G132T alepo c.G132C
c.T258G or c.T258A 
c.T258G alepo c.T258A
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c.148T>C or c.150C>G or c.150C>A 
c.148T>C alepo c.150C>G alepo c.150C>A
c.140G>A or 141G>A 
c.140G>A alepo 141G>A
UNKNOWN (del Exon3?) 
NEZNÁME (del Exon3?)
c.G471C or c.G471T 
c.G471C alepo c.G471T
In the ERT-experienced trial, following 18 months of treatment with Galafold there was a statistically significant decrease in LVMi (p< 0.05). 
V skúšaní u pacientov, ktorí mali skúsenosti s ERT, sa po 18 mesiacoch liečby liekom Galafold pozorovalo štatisticky významné zníženie LVMi (p <
c.1074G>T or c.1074G>C 
c.1074G>T alepo c.1074G>C
c.T881G or c.T881A 
c.T881G alepo c.T881A
c.243G>T or c.243G>C 
c.243G>T alepo c.243G>C
c.561G>T or c.561G>A or c.561G>C 
c.561G>T alepo c.561G>A alepo c.561G>C
c.132G>T or c.132G>C 
c.132G>T alepo c.132G>C
92.9 g/m for the ERT arm and the mean change from baseline in LVMi at Month 18 was -6.6 (95% CI: -11.0, -2.1; n=31) for Galafold and -2.0 (95% CI: -11.0, 7.0; n=13) for ERT. 
18. mesiaci predstavovala -6,6 (95 % interval spoľahlivosti: -11,0; -2,1; n=31) pre Galafold a -2,0 (95 % interval spoľahlivosti: -11,0; 7,0; n=13) pre ERT.
c.471G>C or c.471G>T 
c.471G>C alepo c.471G>T
c.124A>C or c.124A>T 
c.124A>C alepo c.124A>T
c.T514A or c.G515C 
c.T514A alepo c.G515C
c.G611A or G612A 
c.G611A alepo G612A
c.C747G or c.C747A 
c.C747G alepo c.C747A
c.612G>T or c.612G>C 
c.612G>T alepo c.612G>C
c.T148C or c.C150G or c.C150A F50L 
c.T148C alepo c.C150G alepo c.C150A
c.228G>C or c.228G>A or c.228G>T 
c.228G>C alepo c.228G>A alepo c.228G>T
c.166T>A or c.167G>C 
c.166T>A alepo c.167G>C
c.960T>G or c.960T>A 
c.960T>G alepo c.960T>A
c.A840T or c.A840C 
c.A840T alepo c.A840C
UNKNOWN (del Exon1_2?) 
NEZNÁME (del Exon1_2?)
Galafold 123 mg hard capsules Migalastat 
Galafold 123 mg tvrdé kapsuly migalastát
c.982G>A or c.982G>C 
c.982G>A alepo c.982G>C
c.337T>C or c.339T>A or c.339T>G 
c.337T>C alepo c.339T>A alepo c.339T>G
c.[138C>G; 153G>T; 167G>T] c.C138G/G153T/G167T 
c.[138C>G; 153G>T; 167G>T]
c.G612T or c.G612C 
c.G612T alepo c.G612C
c.817T>C or c.819T>A or c.819T>G 
c.817T>C alepo c.819T>A alepo c.819T>G
c.T960G or c.T960A 
c.T960G alepo c.T960A
UNKNOWN (del Exon5_7?) 
NEZNÁME (del Exon5_7?)
Galafold 123 mg hard capsules 
Galafold 123 mg tvrdé kapsuly
c.C897G or c.C897A 
c.C897G alepo c.C897A
c.G1074T or c.G1074C 
c.G1074T alepo c.G1074C
c.G567C or c.G567T 
c.G567C alepo c.G567T
c.G144T or c.G144C 
c.G144T alepo c.G144C
Paraesthesia Dizziness Hypoaesthesia 
Poruchy nervového systému bolesť hlavy parestézia závraty hypoestézia
c.816C>A or c.816C>G 
c.816C>A alepo c.816C>G
c.G1081A or c.G1081C 
c.G1081A alepo c.G1081C
c.A588T or c.A588C 
c.A588T alepo c.A588C
c.G243T or c.G243C 
c.G243T alepo c.G243C
c.G103C or c.G103A 
c.G103C alepo c.G103A
The first Phase 3 trial (ATTRACT) was a randomised open-label active comparator trial that evaluated the efficacy and safety of Galafold compared to enzyme replacement therapy (ERT) (agalsidase beta, agalsidase alfa) in 52 male and female patients with Fabry disease who were receiving ERT prior to trial entry and who have amenable mutations (ERT-experienced trial). 
Prvé skúšanie fázy 3 (ATTRACT) bolo randomizované otvorené skúšanie s aktívnym porovnávacím liekom, v ktorom sa hodnotila účinnosť a bezpečnosť lieku Galafold v porovnaní s enzýmovou substitučnou liečbou (ERT)
c.881T>G or c.881T>A 
c.881T>G alepo c.881T>A
c.153G>A or c.153G>T or c.153G>C 
c.153G>A alepo c.153G>T alepo c.153G>C
c.T784A or c.T784C 
c.T784A alepo c.T784C
LOT: 
Tel.:
c.T154A or c.G155C 
c.T154A alepo c.G155C
c.144G>T or c.144G>C 
c.144G>T alepo c.144G>C
c.258T>G or c.258T>A 
c.258T>G alepo c.258T>A
c.707G>A or c.708G>A 
c.707G>A alepo c.708G>A
c.G734A or c.G735A 
c.G734A alepo c.G735A
c.1081G>A or c.1081G>C 
c.1081G>A alepo c.1081G>C
C]-migalastat HCl revealed that 99% of the radiolabeled dose recovered in plasma was comprised of unchanged migalastat (77%) and 3 dehydrogenated O-glucuronide conjugated metabolites, M1to M3 (13%). 
%) a z troch dehydrogenovaných metabolitov konjugovaných s O-glukuronidom, M1 až M3 (13
c.394G>A or c.394G>C 
c.394G>A alepo c.394G>C
Galafold is not recommended for use in patients with severe renal insufficiency, defined as estimated GFR less than 30 mL/min/1.73m 
Galafold sa neodporúča používať u pacientov so závažnou renálnou insuficienciou definovanou ako odhadnutá hodnota GFR nižšia ako 30 ml/min/1,73 m2 (pozri časť 5.2).
c.G216A or c.G216T or c.G216C M72I 
c.G216A alepo c.G216T alepo c.G216C
c.G831T or c.G831C 
c.G831T alepo c.G831C
c.G830A or c.G831A 
c.G830A alepo c.G831A
The mean change from baseline in LVMi at Month 18 to 24 in patients with left ventricular hypertrophy at baseline (females with baseline LVMi > 95 g/m or males with baseline LVMi > 115 g/m 2) was -18.6 (95% CI: -38.2, 1.0; n=8). 
Priemerná zmena LVMi od začiatku do 18. - 24. mesiaca u pacientov s hypertrofiou ľavej komory na začiatku (ženy s LVMi na začiatku > 95 g/m2 alebo muži s LVMi na začiatku > 115 g/m2) bola -18,6
c.G140A or G141A 
c.G140A alepo G141A
Protein Sequence change c.744delT 
Zmena sekvencie proteínov c.214delA
c.897C>G or c.897C>A 
c.897C>G alepo c.897C>A
c.141G>C or c.141G>T 
c.141G>C alepo c.141G>T
c.G609C or c.G609T 
c.G609C alepo c.G609T
Blood Creatine Phosphokinase increased 
Laboratórne a funkčné vyšetrenia zvýšená hladina kreatínfosfokinázy v krvi zvýšená telesná hmotnosť
c.G71A or c.G72A 
c.G71A alepo c.G72A
c.860G>A or c.861G>A 
c.860G>A alepo c.861G>A
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c.540G>C or c.540G>T 
c.540G>C alepo c.540G>T
c.103G>C or c.103G>A 
c.103G>C alepo c.103G>A
c.567G>C or c.567G>T 
c.567G>C alepo c.567G>T
c.609G>C or c.609G>T 
c.609G>C alepo c.609G>T
c.G141C or c.G141T 
c.G141C alepo c.G141T
c.830G>A or c.831G>A 
c.830G>A alepo c.831G>A
The mean annualised rate of change from baseline in eGFRCKD-EPI in patients treated for 30 months with Galafold was -1.72 mL/min/1.73 m 
Priemerná anualizovaná miera zmeny eGFRCKD-EPI u pacientov liečených počas tridsiatich mesiacov liekom Galafold od začiatku liečby bola -1,72 ml/min/1,73 m2 (95 % IS: -2,653; -0,782; n=31).
c.G860A or c.G861A 
c.G860A alepo c.G861A
c.734G>A or c.735G>A 
c.734G>A alepo c.735G>A
Headache and dizziness were the most common adverse reactions reported at doses of Galafold of up to 1250 mg and 2000 mg, respectively. 
Najčastejšie nežiaduce reakcie hlásené pri užívaní lieku Galafold v dávkach do 1 250 mg a 2 000 mg boli bolesť hlavy, závraty, v uvedenom poradí.
c.831G>T or c.831G>C 
c.831G>T alepo c.831G>C
Migalastat binding stabilizes these mutant forms of -Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes. 
v endoplazmatickom retikule a uľahčuje ich náležitú migráciu do lyzozómov.
c.G861C or c.G861T 
c.G861C alepo c.G861T
c.T859C or c.T859A 
c.T859C alepo c.T859A
c.486G>C or c.486G>T 
c.486G>C alepo c.486G>T
c.288G>A or c.288G>T or c.288G>C 
c.288G>A alepo c.288G>T alepo c.288G>C
c.784T>A or c.784T>C 
c.784T>A alepo c.784T>C
c.108G>C or c.108G>T 
c.108G>C alepo c.108G>T
c.C159G or c.C159A 
c.159C>G alepo c.159C>A c.C159G alepo c.C159A
c.514T>A or c.515G>C 
c.514T>A alepo c.515G>C
c.154T>A or c.155G>C 
c.154T>A alepo c.155G>C
c.T817C or c.T819A or c.T819G F273L 
c.T817C alepo c.T819A alepo c.T819G
c.G394A or c.G394C 
c.G394A alepo c.G394C
c.T667A or c.G668C 
c.T667A alepo c.G668C
During the open-label extension, statistically significant (p<0.05) improvements from baseline were observed in the diarrhoea and indigestion domains, with a trend of improvement in the constipation domain. 
0,05) zlepšenia oproti začiatku, pokiaľ ide o hnačku a trávenie a pozoroval sa trend zlepšenia, pokiaľ ide o zápchu.
c.859T>C or c.859T>A 
c.859T>C alepo c.859T>A
c.T834G or c.T834A 
c.T834G alepo c.T834A
After an average of 3.4 years of treatment, the mean annualised rate of change in eGFRCKD-EPI was -0.74 mL/min/1.73 m 
Po priemerne 3,4 roka liečby bola priemerná anualizovaná miera zmeny eGFRCKD-EPI -0,74 ml/min/1,73 m2 (95 % IS: -1,89; 0,40; n=41).
UNKNOWN (del Exon3_7?) 
NEZNÁME (del Exon3_7?)
c.588A>T or c.588A>C 
c.588A>T alepo c.588A>C
c.1065C>A or c.1065C>G c.C1065A or c.C1065G 
M353R c.1065C>A alepo c.1065C>G c.C1065A alepo c.C1065G
c.A868C or c.A868T 
c.A868C alepo c.A868T
c.T894G or c.T894A 
c.T894G alepo c.T894A
c.G785A or c.G786A 
c.G785A alepo c.G786A
c.G108C or c.G108T 
c.G108C alepo c.G108T
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c.G486C or c.G486T 
c.G486C alepo c.G486T
c.834T>G or c.834T>A 
c.834T>G alepo c.834T>A
c.71G>A or c.72G>A 
c.71G>A alepo c.72G>A
If more than 12 hours has passed you should resume taking Galafold at the next planned dosing day and time according to your every other day dosing schedule.. Do not take two capsules to make up for your missed dose. 
Ak uplynie viac ako 12 hodín, pokračujte v užívaní lieku Galafold v najbližší plánovaný deň a čas užitia dávky v súlade s dávkovacím režimom každý druhý deň.
c.T280A or c.G281C 
c.T280A alepo c.G281C
After follow up in the OLE, the mean change from baseline in LVMi in patients with left ventricular hypertrophy at baseline at Month 36 was -30.0 (95%) CI: -57.9, -2.2; n=4) and at Month 48 
Po sledovaní v OLE bola priemerná zmena LVMi od začiatku do 36. mesiaca u pacientov s hypertrofiou ľavej komory na začiatku -30,0 (95 % IS: -57,9; -2,2; n=4) a do 48. mesiaca 33,1 (IS: -60,9; -5,4; n=4).
See package leaflet for further instructions. 
Ďalšie pokyny sa nachádzajú v písomnej informácii pre používateľa.
c.785G>A or c.786G>A 
c.785G>A alepo c.786G>A
UNKNOWN (del Exon2_3?) 
NEZNÁME (del Exon2_3?)
c.978G>C or c.978G>T 
c.978G>C alepo c.978G>T
c.708G>C or c.708G>T 
c.708G>C alepo c.708G>T
c.702T>G or c.702T>A 
c.702T>G alepo c.702T>A
c.T337C or c.T339A or c.T339G F113L 
c.T337C alepo c.T339A alepo c.T339G
c.G707A or c.G708A 
c.G707A alepo c.G708A
c.837G>C or c.837G>T 
c.837G>C alepo c.837G>T
If you are planning to have a baby, ask your doctor, pharmacist, or nurse for advice. 
Ak plánujete otehotnieť, poraďte sa so svojím lekárom, lekárnikom alebo zdravotnou sestrou.
c.894T>G or c.894T>A 
c.894T>G alepo c.894T>A
c.720G>C or c.720G>T 
c.720G>C alepo c.720G>T
c.747C>G or c.747C>A 
c.747C>G alepo c.747C>A
c.870G>A or c.870G>C or c.870G>T 
c.870G>A alepo c.870G>C alepo c.870G>T
c.888G>A or c.888G>T or c.888G>C 
c.888G>A alepo c.888G>T alepo c.888G>C
The change from baseline to Month 18 in LVMi (g/m 2) in patients with left ventricular hypertrophy (females with baseline LVMi > 95 g/m and males with baseline LVMi > 115 g/m2) was -8.4 (95% CI: -15.7, 2.6; n=13) for migalastat and 4.5 (95% CI: -10.7, 18.4; n=5) for ERT. After 30 months treatment with Galafold, the mean change from baseline in LVMi was -3.8 (95% CI: -8.9, 1.3; n=28) and the mean change from baseline in LVMi in patients with left ventricular hypertrophy at baseline was -10.0 (95% CI: -16.6, -3.3; n=10). 
18. mesiaca u pacientov s hypertrofiou ľavej komory (ženy s LVMi na začiatku > 95 g/m2 a muži s LVMi na začiatku > 115 g/m2) bola -8,4 (95 % IS: -15,7; 2,6; n=13) pre migalastát a 4,5 (95 % IS: -10,7; 18,4; n=5) pre ERT.
c.[790G>T; 805G>A] 
Zmena sekvencie proteínov c.[790G>T; 805G>A]
c.936G>T or c.936G>C 
c.936G>T alepo c.936G>C
c.T402G or c.T402A 
c.T402G alepo c.T402A
c.G772C or c.G772A 
c.G772C alepo c.G772A
c.G729C or c.G729T 
c.G729C alepo c.G729T
c.G837C or c.G837T 
c.G837C alepo c.G837T
c.T702G or c.T702A 
c.T702G alepo c.T702A
c.G561T or c.G561A or c.G561C M187I 
c.G561T alepo c.G561A alepo c.G561C
c.G978C or c.G978T 
c.G978C alepo c.G978T
c.A886T or c.A886C 
c.A886T alepo c.A886C
c.1095T>A or c.1095T>G c.T1095A or c.T1095G 
R363P c.1095T>A alepo c.1095T>G c.T1095A alepo c.T1095G Y365X c.1115T>A
c.706T>C or c.706T>A 
c.706T>C alepo c.706T>A
c.102T>G or c.102T>A 
c.102T>G alepo c.102T>A
c.963G>C or c.963G>T 
c.963G>C alepo c.963G>T
c.840A>T or c.840A>C 
c.840A>T alepo c.840A>C
c.T706C or c.T706A 
c.T706C alepo c.T706A
c.G720C or c.G720T 
c.G720C alepo c.G720T
c.G708C or c.G708T 
c.G708C alepo c.G708T
c.667T>A or c.668G>C 
c.667T>A alepo c.668G>C
c.A712C or c.T714A or c.T714G S238R 
c.A712C alepo c.T714A alepo c.T714G
c.T70C or c.T70A 
c.T70C alepo c.T70A
c.729G>C or c.729G>T 
c.729G>C alepo c.729G>T
c.402T>G or c.402T>A 
c.402T>G alepo c.402T>A
c.T102G or c.T102A 
c.T102G alepo c.T102A
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c.G228C or c.G228A or c.G228T M76I 
c.G228C alepo c.G228A alepo c.G228T
c.G126A or c.G126C or c.G126T M42I 
c.G126A alepo c.G126C alepo c.G126T
c.C279G or c.C279A 
c.279C>G alepo c.279C>A c.C279G alepo c.C279A
c.886A>T or c.886A>C 
c.886A>T alepo c.886A>C
Diarrhoea Nausea 
Psychické poruchy depresia
c.868A>C or c.868A>T 
c.868A>C alepo c.868A>T
c.72G>C or c.72G>T 
c.72G>C alepo c.72G>T
c.G778C or c.G778A 
c.G778C alepo c.G778A
c.678G>T or c.678G>C 
c.678G>T alepo c.678G>C
Do not take Galafold if you: 
Neužívajte Galafold
Table 1: Adverse reactions with Galafold in clinical trials System Organ Class 
Galafold hlásené v klinických skúšaniach
c.70T>C or c.70T>A 
c.70T>C alepo c.70T>A
c.G288A or c.G288T or c.G288C M96I 
c.G288A alepo c.G288T alepo c.G288C
c.G153A or c.G153T or c.G153C M51I 
c.G153A alepo c.G153T alepo c.G153C
c.772G>C or c.772G>A 
c.772G>C alepo c.772G>A
Patient-Reported Outcome - Gastrointestinal Symptoms Rating Scale In the ERT-naïve trial, analyses of the Gastrointestinal Symptoms Rating Scale demonstrated that treatment with Galafold was associated with statistically significant (p<0.05) improvements versus placebo from baseline to month 6 in the diarrhoea domain, and in the reflux domain for patients with symptoms at baseline. 
V skúšaní u pacientov, ktorí nedostávali ERT, analýzy stupnice hodnotenia gastrointestinálnych symptómov preukázali, že liečba liekom Galafold sa spájala so štatisticky významnými (p <
c.786G>C or c.786G>T 
c.786G>C alepo c.786G>T
Amicus Therapeutics Europe Limited Teл. 
Therapeutics Europe Limited Tél/Tel:
c.1023A >C or c.1023A>T 
>C alepo c.1023A>T
c.G678T or c.G678C 
c.G678T alepo c.G678C
c.G786C or c.G786T 
c.G786C alepo c.G786T
: 00800 111 3214 имейл: MedInfo@amicusrx.com 
Limited Teл.: 00800 111 3214 имейл: MedInfo@amicusrx.com
1.478; n=34) in the Galafold group compared to -1.03 mL/min/1.73 m 
Priemerná anualizovaná miera zmeny eGFRCKD-EPI bola -0,40 ml/min/1,73 m2 (95 % IS: -2,272; 1,478; n=34) v skupine užívajúcej liek Galafold v porovnaní s -1,03 ml/min/1,73 m2
c.778G>C or c.778G>A 
c.778G>C alepo c.778G>A
c.G888A or c.G888T or c.G888C M296I 
c.G888A alepo c.G888T alepo c.G888C
Amicus Therapeutics Europe Limited Tlf: 800 13837 e-post: MedInfo@amicusrx.com 
Limited Tlf: 800 13837 e-post: MedInfo@amicusrx.com
c.G72C or c.G72T 
c.G72C alepo c.G72T
Protein Sequence change c.290delC 
Zmena sekvencie proteínov c.722delG
c.T139C or c.T139A 
c.139T>C alepo c.139T>A c.T139C alepo c.T139A
• Protein in the urine 
• bielkovina v moči
You should not take Galafold if you are also receiving enzyme replacement therapy. 
Ak dostávate aj enzýmovú substitučnú liečbu, neužívajte liek Galafold.
c.G963C or c.G963T 
c.G963C alepo c.G963T
c.1018T>C or c.1018T>A c.T1018C or c.T1018A 
V339G c.1018T>C alepo c.1018T>A
c.[1067G>A; 1078G>C] 
N355K c.[1067G>A; 1078G>C]
c.G870A or c.G870C or c.G870T M290I 
c.G870A alepo c.G870C alepo c.G870T
c.G936T or c.G936C 
c.G936T alepo c.G936C
c.A442C or c.T444A or c.T444G S148R 
c.A442C alepo c.T444A alepo c.T444G
c.1019G>A or c.1020G>A c.G1019A or c.G1020A 
W340R c.1019G>A alepo c.1020G>A
• Sensation of spinning 
• pocit nevoľnosti
• Feeling sick (nausea) 
• pocit točenia (vertigo)
Amicus Therapeutics Europe Limited Tel: 1800 936 230 e-mail: MedInfo@amicusrx.com 
Limited Tel: 1800 936 230 e-mail: MedInfo@amicusrx.com
c.552T>A or c.552T>G 
c.552T>A alepo c.552T>G c.T552A alepo c.T552G
c.519C>A or c.519C>G 
c.519C>A alepo c.519C>G c.C519A alepo c.C519G
c.G284A or c.G285A 
c.284G>A alepo c.285G>A c.G284A alepo c.G285A
c.C207A or c.C207G 
c.207C>A alepo c.207C>G c.C207A alepo c.C207G
c.412G>A or c.412G>C 
c.412G>A alepo c.412G>C c.G412A alepo c.G412C
Protein sequence change c.126G>A or c.126G>C or c.126G>T 
c.126G>A alepo c.126G>C alepo c.126G>T
Amicus Therapeutics Europe Limited Puh/Tel: 0800 917 780 sähköposti/e-mail: MedInfo@amicusrx.com 
Therapeutics Europe Limited Tél/Tel: 0800 79245 e-mail: MedInfo@amicusrx.com
c.T676C or c.T676A 
c.676T>C alepo c.676T>A c.T676C alepo c.T676A
c.1034C>G or c.1034C>A c.C1034G or c.C1034A 
L344P c.1034C>G alepo c.1034C>A
Protein Sequence change c.1202C>G or c.1202C>A c.C1202G or c.C1202A 
W399S c.1202C>G alepo c.1202C>A
Amicus Therapeutics Europe Limited Tel.: 06 800 21202 e-mail: MedInfo@amicusrx.com 
Limited Tel.: 06 800 21202 e-mail: MedInfo@amicusrx.com
c.G131A or c.G132A 
c.131G>A alepo c.132G>A c.G131A alepo c.G132A
c.C453G or c.C453A 
c.453C>G alepo c.453C>A c.C453G alepo c.C453A
Amicus Therapeutics Europe Limited Tel: 0800 222 452 e-pošta: MedInfo@amicusrx.com 
Limited Tel: 0800 222 452 e-pošta: MedInfo@amicusrx.com
c.456C>A or c.456C>G 
c.456C>A alepo c.456C>G c.C456A alepo c.C456G
• Diarrhoea 
• bolesti
c.[644A>G; 811G>A; 937G>T] c.A644G/G811A/G937T 
c.[644A>G; 811G>A; 937G>T]
Musculoskeletal and connective tissue disorders 
Poruchy kostrovej a svalovej sústavy a spojivového tkaniva svalové kŕče myalgia tortikolis bolesti končatín
c.T241C or c.T241A 
c.241T>C alepo c.241T>A c.T241C alepo c.T241A
c.C233G or c.C233A 
c.233C>G alepo c.233C>A c.C233G alepo c.C233A
c.465T>A or c.465T>G 
c.465T>A alepo c.465T>G c.T465A alepo c.T465G
C]-migalastat hydrochloride in the concentration range between 1 and 
Väzba na plazmatické proteíny po podaní [14C]-migalastátiumchloridu v rozsahu koncentrácií 1 - 100 M bola nezistiteľná.
Amicus Therapeutics Europe Limited Tel.: 0080 012 15475 e-mail: MedInfo@amicusrx.com 
Limited Tel.: 0080 012 15475 e-mail: MedInfo@amicusrx.com
g.6009_9741del 
NEZNÁME (del Exon3 a g.6009_9741del
c.966C>A or c.966C>G 
c.966C>A alepo c.966C>G c.C966A alepo c.C966G
c.T610C or c.T610A 
c.610T>C alepo c.610T>A c.T610C alepo c.T610A
Amicus Therapeutics Europe Limited Tel: 800 62674 e-mail: MedInfo@amicusrx.com 
Limited Tel: 800 62674 e-mail: MedInfo@amicusrx.com
Amicus Therapeutics Europe Limited Tel.: 0800 81794 e-pošta: MedInfo@amicusrx.com 
Limited Tel.: 0800 81794 e-pošta: MedInfo@amicusrx.com
c.1046G>A or c.1047G>A c.G1046A or c.G1047A 
c.G1046A alepo c.G1047A
c.1045T>C or c.1045T>A c.T1045C or c.T1045A 
A348P c.1045T>C alepo c.1045T>A
There was no detectable plasma protein binding following administration of [ 
rádioaktívne označenej dávky zistenej v plazme pozostávalo z nezmeneného migalastátu (77
c.408T>A or c.408T>G 
c.408T>A alepo c.408T>G c.T408A alepo c.T408G
BLISTER SLEEVE 
Č. šarže
Patients randomised to Galafold in Stage 1 demonstrated statistically significant greater reduction (±SEM) in mean interstitial capillary GL-3 deposition (-0.25±0.10; -39%) at month 6 compared to placebo (+0.07 ± 0.13; +14%) (p=0.008). 
0,10; -39 %) v šiestom mesiaci v porovnaní s placebom (+0,07 ±
c.861G>C or c.861G>T 
Zmena sekvencie proteínov c.861G>C alepo c.861G>T
c.T484C or c.T484A 
c.484T>C alepo c.484T>A c.T484C alepo c.T484A
c.648T>A or c.648T>G 
c.648T>A alepo c.648T>G c.T648A alepo c.T648G
c.525C>G or c.525C>A 
Zmena sekvencie proteínov c.525C>G alepo c.525C>A
c.G485A or c.G486A 
c.485G>A alepo c.486G>A c.G485A alepo c.G486A
c.789T>A or c.789T>G 
c.789T>A alepo c.789T>G c.T789A alepo c.T789G
c.C666A or c.C666G 
c.666C>A alepo c.666C>G c.C666A alepo c.C666G
c.3G>A or c.3G>T or c.3G>C c.G3A or c.G3T or c.G3C 
c.3G>A alepo c.3G>T alepo c.3G>C
c.798T>A or c.798T>G 
c.798T>A alepo c.798T>G c.T798A alepo c.T798G
c.T369G or c.T369A 
c.369T>G alepo c.369T>A c.T369G alepo c.T369A
c.439G>A or c.439G>C 
c.439G>A alepo c.439G>C c.G439A alepo c.G439C
c.242G>A or c.243G>A 
c.242G>A alepo c.243G>A c.G242A alepo c.G243A
g.6547_9783del 
NEZNÁME (del Exon3 a g.6547_9783del
: 80 253 262 e-mail: MedInfo@amicusrx.com 
Limited Tlf.: 80 253 262 e-mail: MedInfo@amicusrx.com
c.687T>A or c.687T>G 
c.687T>A alepo c.687T>G c.T687A alepo c.T687G
Abdominal pain Constipation Dry mouth 
Poruchy gastrointestinálneho traktu hnačka nauzea bolesť brucha zápcha sucho v ústach nutkanie na stolicu dyspepsia
Gastrointestinal disorders 
Výsledok udávaný pacientmi - stupnica hodnotenia gastrointestinálnych symptómov
Amicus Therapeutics Europe Limited Tlf. 
Amicus Therapeutics, UK Limited Tel:
UNKNOWN IVS6-10G>A; IVS6-22C>T c.1000-10G>A/1000-22C>T UNKNOWN 
c.1000-10G>A/1000-22C>T
Slovenská republika Amicus Therapeutics Europe Limited Tel.: 0800 002 437 e-mail: MedInfo@amicusrx.com 
Limited Tel.: 0800 002 437 e-mail: MedInfo@amicusrx.com
EMA/509804/2018 EMEA/H/C/004866 
EMEA/H/C/004866
Further information on Halimatoz can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/European public assessment reports. 
Halimatoz sa nachádzajú na webovej stránke agentúry: ema.europa.eu/Find medicine/Human medicines/European public assessment reports.
The proportion of patients who had at least a 75% reduction in symptoms after 16 weeks of treatment was 68% with Halimatoz and 63% with Humira. 
Podiel pacientov, u ktorých došlo k zmierneniu príznakov najmenej o 75 % po 16 týždňoch liečby bol 68 % u pacientov liečených liekom Hyrimoz a 63 % u pacientov liečených liekom Humira.
This means that Halimatoz is highly similar to another biological medicine (the ‘reference medicine') that is already authorised in the EU. 
To znamená, že liek Halimatoz je vysoko podobný inému biologickému lieku (tzv. referenčnému lieku), ktorý je už v EÚ povolený.
In addition, Halimatoz was as effective as Humira in a study involving 465 patients with moderate or severe plaque psoriasis. 
Halimatoz bol okrem toho rovnako účinný ako liek Humira v štúdii zahŕňajúcej 465 pacientov so stredne závažnou alebo závažnou ložiskovou psoriázou.