natgillin/translations-raw huggingface dataset:
Do not eat food at least 2 hours before and 2 hours after taking your medicine to give a minimum 4 hours fast.
Nejedzte najmenej 2 hodiny pred užitím a 2 hodiny po užití lieku, aby sa dosiahol minimálne 4hodinový stav nalačno.
Elderly population No dosage adjustment is required based on age (see section 5.2).
Starší pacienti Úprava dávkovania na základe veku nie je potrebná (pozri časť 5.2).
Skin and subcutaneous tissue disorders
Poruchy kože a podkožného tkaniva vyrážka pruritus
Almac Pharma Services (Ireland) Limited Finnabair Industrial Estate Dundalk, Co. Louth
Limited Finnabair Industrial Estate Dundalk, Co.
In a single dose study with Galafold in non-Fabry subjects with varying degrees of renal insufficiency, exposures were increased by 4.3-fold in subjects with severe renal impairment (GFR < 30 mL/min/1.73 m
V štúdii skúmajúcej podávanie jednej dávky lieku Galafold u jedincov bez Fabryho choroby s rôznymi stupňami renálnej nedostatočnosti sa expozícia zvýšila 4,3-násobne u jedincov so závažným poškodením funkcie obličiek (GFR < 30 ml/min/1,73 m2).
What Galafold looks like and contents of the pack Opaque, blue, and white hard capsules, marked with “A1001” in black ink, containing white to pale brown powder.
Nepriehľadné modro-biele tvrdé kapsuly označené čiernym nápisom „A1001“, obsahujúce biely až svetlohnedý prášok.
Weight increased
• zvýšenie telesnej hmotnosti
The second OLE trial (AT1001-042) included patients that both transferred from OLE study AT1001-041 and directly from Phase 3 study ATTRACT, and is ongoing.Renal Function
Do druhého OLE skúšania (AT1001-042) boli zaradení pacienti, ktorí prešli zo skúšania s OLE AT1001-041 aj priamo zo skúšania ATTRACT v 3. fáze, a toto skúšanie stále prebieha.
If you stop taking Galafold Do not stop taking this medicine without talking to your doctor.
Neprestávajte užívať tento liek bez toho, aby ste sa poradili so svojím lekárom.
PUSH capsule through the foil 4.
VYTIAHNITE kartu na pravej strane, 3. PRETLAČTE kapsulu cez fóliu, 4.
c.760-762delGTT or c.761-763del c.760_762delGTT or c.761_763del p.V254del
c.760-762delGTT alepo c.761763del c.760_762delGTT alepo c.761_763del p.V254del
Fabry disease-causing mutations in the GLA gene result in a deficiency of the lysosomal enzyme -galactosidase A (-Gal A) that is required for glycosphingolipid substrate (e.g., GL-3, lyso-Gb3) metabolism.
Mutácie v géne GLA zapríčinené Fabryho chorobou vedú k nedostatku lyzozomálneho enzýmu -galaktozidázy A (-Gal A), ktorý je potrebný pre metabolizmus glykosfingolipidového substrátu (napr.
What Galafold contains - The active substance is migalastat.
Čo Galafold obsahuje - Liečivo je migalastát.
The absolute bioavailability (AUC) for a single oral 150 mg migalastat hydrochloride dose or a single 2-hour 150 mg intravenous infusion was approximately 75%.
Absolútna biologická dostupnosť (AUC) jednorazovej perorálnej 150 mg dávky migalastátiumchloridu alebo jednorazovej dvojhodinovej 150 mg intravenóznej infúzie dosahovala približne 75 %. Po jednej perorálnej 150 mg dávke roztoku migalastátiumchloridu bola maximálna plazmatická koncentrácia dosiahnutá približne za tri hodiny.
c.963_964GG>CA
c.963_964GG>
g.6736_11545del c.370-533_c.1290+277del
NEZNÁME (del Exon3 a g.6736_11545del c.370-533_c.1290+277del
If more than 12 hours has passed the patient should resume taking Galafold at the next planned dosing day and time according to the every other day dosing schedule.
Ak uplynie viac ako 12 hodín, pacient má pokračovať v užívaní lieku Galafold v najbližší plánovaný deň a čas užitia dávky v súlade s dávkovacím režimom každý druhý deň.
Amicus Therapeutics Europe Limited
Amicus Therapeutics Europe
c.A1023C or c.A1023T
c.A1023C alepo c.A1023T
The baseline values were 95.3 g/m for the Galafold arm and
Východiskové hodnoty boli 95,3 g/m2 pre rameno Galafold a 92,9 g/m2 pre rameno ERT a priemerná zmena východiskového LVMi v
• Sudden need to defecate
• náhla potreba vyprázdniť sa
p.M42Tfs*74 c.134_138delTGCACinsGCTCG c.134_138delTGCACinsGCTCG L45R/H46S c.147_148insCCC
p.M42Tfs*74 c.134_138delTGCACinsGCTCG c.134_138delTGCACinsGCTCG
Galafold contains the active substance migalastat.
Galafold obsahuje liečivo migalastát.
(proteinuria)
(proteinúria)
g.941_5845del p.?
g.941_5845del
In the ERT- naïve trial and open-label extension, renal function remained stable for up to 5 years of treatment with Galafold.
V skúšaní u pacientov, ktorí nedostávali ERT, a v otvorenom predĺženom skúšaní ostala funkcia obličiek stabilná do 5 rokov liečby liekom Galafold.
C]-migalastat hydrochloride revealed that approximately 77% of the radiolabeled dose was recovered in urine of which 55% of was excreted as unchanged migalastat and 4% as combined metabolites M1, M2 and M3.
Pri farmakokinetickom skúšaní u zdravých dobrovoľníkov - mužov so 150 mg [14C]-migalastátiumchloridu sa zistilo, že približne 77 % rádioaktívne označenej dávky sa nachádzalo v moči, z čoho 55 % dávky sa vylúčilo ako nezmenený migalastát a 4 % ako kombinované metabolity M1, M2 a M3. Približne 5 % celkovej rádioaktivity vzorky tvorili nezistené zložky.
Qualitative reductions in GL-3 levels were observed in multiple renal cell types: podocytes, mesangial cells, and glomerular endothelial cells, respectively, over 12 months of treatment with Galafold.
Galafold sa pozorovali kvalitatívne zníženia hladiny GL-3 vo viacerých typoch renálnych buniek: v podocytoch, mezangiálnych bunkách a v glomerulárnych endoteliálnych bunkách.
Galafold should not be taken on 2 consecutive days.
Galafold sa nemá užívať dva po sebe nasledujúce dni.
Amicus Therapeutics Europe Limited Tel: 0800 005 475
Limited Tel: 0800 005 475
Fertility in women It is not yet known if this medicine affects fertility in women.
Dosiaľ nie je známe, či tento liek ovplyvňuje plodnosť u žien.
Respiratory, thoracic, and mediastinal disorders
Poruchy dýchacej sústavy, hrudníka a mediastína dyspnoe epistaxa
g.10237_11932del/11933_12083i nv/12084_12097del
g.10237_11932del/11933_12083i nv/12084_12097del g.>5.5kbdel až 3UTR
Protein Sequence change c.1A>C or c.1A>T
Zmena sekvencie proteínov c.1A>C alepo c.1A>T
The mean extent of exposure to the marketed dose of Galafold 123 mg QOD in patients completing study AT1001-041 was 3.57 (±1.23) years (n=85).
Stredná hodnota rozsahu expozície dávke 123 mg každý druhý deň lieku Galafold uvedenej na trh u pacientov dokončujúcich skúšanie AT1001-041 bola 3,57 (±
c.A504C or c.A504T
c.A504C alepo c.A504T
• Painful stiff neck
• bolestivá stuhnutosť krku (tortikolis)
c.A1C or c.A1T
c.A1C alepo c.A1T
The frequency of events in patients treated with Galafold over 30 months (32%) was similar to the 18 month period.
Frekvencia udalostí u pacientov liečených liekom Galafold počas tridsiatich mesiacov (32
c.1196G>A or c.1197G>A c.G1196A or c.G1197A
E398A c.1196G>A alepo c.1197G>A c.G1196A alepo c.G1197A
Hepatic impairment No dosage adjustment of Galafold is required in patients with hepatic impairment (see section 5.2).
U pacientov s poruchou funkcie pečene nie je potrebná úprava dávkovania lieku Galafold (pozri časť 5.2).
Each capsule contains migalastat hydrochloride equivalent to 123 mg migalastat - The other ingredients are: Capsule contents: Pregelatinised maize starch and magnesium stearate Capsule shell: Gelatin, titanium dioxide, and indigotine Printing ink: Shellac, black iron oxide, and potassium hydroxide
Každá kapsula obsahuje migalastátumchlorid, čo zodpovedá 123 mg migalastátu - Ďalšie zložky sú: Obsah kapsuly: predželatínovaný kukuričný škrob a magnéziumstearát
c.A924T or c.A924C
c.A924T alepo c.A924C
Patients randomised to placebo in Stage 1 and switched to Galafold at month 6 (Stage 2) also demonstrated statistically significant decreases in interstitial capillary GL-3 inclusions at month 12 (-0.33±0.15; -58%) (p=0.014).
Pacienti randomizovaní na liečbu placebom v 1. štádiu, ktorí prešli na liek Galafold v šiestom mesiaci (2. štádium) tiež preukázali štatisticky významné zníženia inklúzií GL-3 v intersticiálnej oblasti kapilár v 12. mesiaci (-0,33 ±
Galafold has not been studied in patients with Fabry disease who have a GFR less than 30 mL/min/1.73 m2.
Galafold sa neskúmal u pacientov s Fabryho chorobou, ktorí majú odhadnutú hodnotu GFR nižšiu ako 30 ml/min/1,73 m2.
c.T1147C or c.C1149G or c.C1149A
c.T1147C alepo c.C1149G alepo c.C1149A
c.C816A or c.C816G
c.C816A alepo c.C816G
c.504A>C or c.504A>T
c.504A>C alepo c.504A>T
c.712A>C or c.714T>A or c.714T>G
c.712A>C alepo c.714T>A alepo c.714T>G
c.442A>C or c.444T>A or c.444T>G
c.442A>C alepo c.444T>A alepo c.444T>G
c.207_369+651del814ins231 UNKNOWN (del Exon2?)
c.207_369+651del814ins231
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
Amicus Therapeutics Europe Limited Sími: 800 7634
Limited Sími: 800 7634
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
Amicus Therapeutics Europe Limited Block 1, Blanchardstown Corporate Park Ballycoolen Road
Limited Block 1, Blanchardstown Corporate
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
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?)
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
Amicus Therapeutics Europe Limited Tel.: 0808 034 288 / 877 309 5040 e-mail: MedInfo@amicusrx.com
Limited Tel.: 0808 034 288 / 877 309 5040 e-mail: MedInfo@amicusrx.com
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
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
Amicus Therapeutics, UK Limited Tel: +44 175 3888 567 / 08 0823 46864 e-mail: MedInfo@amicusrx.com
+44 175 3888 567 / 08 0823 46864 e-mail: MedInfo@amicusrx.com
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).
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
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
Amicus Therapeutics Europe Limited Tfn: 020 795 493 e-post: MedInfo@amicusrx.com
Limited Tfn: 020 795 493 e-post: MedInfo@amicusrx.com
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