natgillin/translations-raw huggingface dataset:
Do not use this medicine after the expiry date, which is stated on the carton and blister after EXP.
Ovaj lijek se ne smije upotrijebiti nakon isteka roka valjanosti navedenog na kutiji i blisteru iza oznake „Rok valjanosti”.
The second Phase 3 trial (FACETS) was a 6-month randomised double-blind placebo-controlled trial (through month 6) with an 18-month open-label period to evaluate the efficacy and safety of Galafold in 50 male and female patients with Fabry disease who were naïve to ERT, or had previously been on ERT and had stopped for at least 6 months and who have amenable mutations (ERT-naïve trial).
Drugo ispitivanje faze 3 (FACETS) bilo je 6-mjesečno randomizirano, dvostruko slijepo i placebom kontrolirano ispitivanje (tijekom 6. mjeseca) s 18-mjesečnim otvorenim razdobljem radi ocjenjivanja djelotvornosti i sigurnosti lijeka Galafold u 50 muških i ženskih bolesnika s Fabryjevom bolešću koji nisu prethodno primali ERT ili koji su prethodno primali ERT i prekinuli su terapiju na najmanje šest mjeseci te koji imaju mutacije koje omogućuju odgovor na liječenje (ispitivanje s bolesnicima koji nisu prethodno primali ERT).
c.1018T>C or c.1018T>A c.T1018C or c.T1018A
c.1018T>C ili c.1018T>A c.T1018C ili c.T1018A
It is not known whether Galafold is secreted in human milk.
Nije poznato luči li se Galafold u majčino mlijeko u ljudi.
Galafold should not be taken on 2 consecutive days.
Galafold se ne smije uzimati dva uzastopna dana.
Within each frequency grouping, adverse reactions are presented in order of decreasing frequency within each System Organ Class.
U okviru svake skupine učestalosti nuspojave su prikazane redoslijedom od veće prema manjoj učestalosti u okviru svakog razreda klasifikacije organskih sustava.
Galafold is not intended for concomitant use with enzyme replacement therapy.
Galafold nije namijenjen za istodobnu primjenu s enzimskom nadomjesnom terapijom.
• Sudden need to defecate
• iznenadna potreba za pražnjenjem crijeva
In patients with amenable mutations, GL-3 levels tended to decrease in urine and in kidney interstitial capillaries.
U bolesnika s mutacijama koje omogućavaju odgovor na liječenje, razine GL-3 imale su tendenciju smanjenja u urinu te u intersticijskim kapilarama bubrega.
• Reduced sense of touch or sensation
• smanjenje osjeta dodira ili osjetljivosti kože (hipoestezija)
(proteinuria)
(proteinurija)
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.
Neprozirne, plave i bijele tvrde kapsule, označene oznakom „A1001” crnom tintom, koje sadrže prašak bijele do svijetlosmeđe boje.
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).
Bolesnici koji su bili randomizirani u skupinu koja je primala Galafold u 1. fazi pokazali su statistički značajno veće smanjenje (±SEM) srednjeg taloženja GL-3 u intersticijskim kapilarama (-0,25 ± 0,10; -39 %) u 6. mjesecu u usporedbi sa skupinom koja je primala placebo (+0,07 ± 0,13; +14 %) (p = 0,008).
Each capsule contains migalastat hydrochloride equivalent to 123 mg migalastat
Svaka kapsula sadrži migalastatklorid koji odgovara 123 mg migalastata
Protein Sequence change c.1202C>G or c.1202C>A c.C1202G or c.C1202A
c.1202C>G ili c.1202C>A c.C1202G ili c.C1202A
Mean annualised rate of change in eGFRCKD-EPI was -0.40 mL/min/1.73 m
Srednja godišnja stopa promjene u eGFRCKD-EPI iznosila je -0,40 mL/min/1,73 m
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%).
99 % radioaktivno označene doze nađene u plazmi sastojalo od neizmijenjenog migalastata (77 %) te od tri dehidrogenirana konjugirana metabolita O-glukuronida M1 do M3 (13 %).
It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on or switched to Galafold.
Savjetuje se da se u bolesnika koji su počeli uzimati migalastat ili koji su prešli na lijek Galafold, periodično (svakih 6 mjeseci) prate bubrežna funkcija, ehokardiografski parametri i biokemijski markeri.
c.1196G>A or c.1197G>A c.G1196A or c.G1197A
c.1196G>A ili c.1197G>A c.G1196A ili c.G1197A
In the ERT- naïve trial, Galafold showed statistically significant reductions in plasma lyso-Gb3 concentrations and kidney interstitial capillary GL-3 inclusions in patients with amenable mutations.
U ispitivanju s bolesnicima koji nisu prethodno primali ERT, Galafold je pokazao statistički značajna smanjenja koncentracija lyso-Gb3 u plazmi te inkluzija GL-3 u intersticijskim bubrežnim kapilarama u bolesnika s mutacijama koje omogućuju odgovor na liječenje.
c.A1C or c.A1T
c.A1C ili c.A1T
Fertility in women It is not yet known if this medicine affects fertility in women.
Još nije poznato utječe li ovaj lijek na plodnost u žena.
c.A1023C or c.A1023T
c.A1023C ili c.A1023T
Treatment with Galafold in Phase 2 pharmacodynamic trials generally resulted in increases in endogenous α-Gal A activity in WBCs, as well as in skin and kidney for the majority of patients.
Liječenje lijekom Galafold u farmakodinamičkim ispitivanjima faze 2 u većine je bolesnika općenito dovelo do povećanja aktivnosti endogenog enzima α-Gal
c.1019G>A or c.1020G>A c.G1019A or c.G1020A
c.1019G>A ili c.1020G>A c.G1019A ili c.G1020A
c.T148C or c.C150G or c.C150A F50L
c.T148C ili c.C150G ili c.C150A F50L
In the ERT-experienced trial, plasma lyso-Gb3 levels slightly increased but remained low in patients with amenable mutations treated with Galafold for the 30 month duration of the study.
U ispitivanju s bolesnicima koji su prethodno primali ERT, razine lyso-Gb3 u plazmi blago su se povećale, ali ostale su niske u bolesnika s mutacijama koje omogućuju odgovor na liječenje koji su liječeni lijekom Galafold u razdoblju od 30 mjeseci trajanja ispitivanja.
How to store Galafold
Kako čuvati Galafold
c.G561T or c.G561A or c.G561C M187I
c.G561T ili c.G561A ili c.G561C M187I
Blood Creatine Phosphokinase increased
Pretrage povišena razina kreatin fosfokinaze u krvi povećana težina
c.T337C or c.T339A or c.T339G F113L
c.T337C ili c.T339A ili c.T339G F113L
c.A712C or c.T714A or c.T714G S238R
c.A712C ili c.T714A ili c.T714G S238R
c.G611A or G612A
c.G611A ili G612A
c.G153A or c.G153T or c.G153C M51I
c.G153A ili c.G153T ili c.G153C M51I
• Sensation of spinning
• osjećaj vrtnje
c.A504C or c.A504T
c.A504C ili c.A504T
If you are planning to have a baby, ask your doctor, pharmacist, or nurse for advice.
Ako planirate imati dijete, obratite se za savjet svom liječniku, ljekarniku ili medicinskoj sestri.
c.T817C or c.T819A or c.T819G F273L
c.T817C ili c.T819A ili c.T819G F273L
In the ERT- naïve trial and open-label extension, renal function remained stable for up to 5 years of treatment with Galafold.
U ispitivanju i otvorenom nastavku ispitivanja s bolesnicima koji nisu prethodno primali ERT, funkcija bubrega ostala je stabilna do 5 godina terapije lijekom Galafold.
Protein Sequence change c.1A>C or c.1A>T
Promjena slijeda u proteinu c.1A>C ili c.1A>T
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.
Farmakokinetičko ispitivanje u zdravih muških dobrovoljaca s dozom od 150 mg C]-migalastatklorida pokazalo je da je otprilike 77 % radioaktivno označene doze pronađeno u urinu od kojih je 55% bilo izlučeno kao neizmijenjeni migalastat, a 4 % kao kombinirani metaboliti M1, M2 i M3. Otprilike 5 % ukupne radioaktivnosti uzorka bilo je od nedodijeljenih komponenata.
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
Srednja godišnja stopa promjene od početne vrijednosti eGFRCKD-EPI u bolesnika liječenih lijekom Galafold u trajanju od 30 mjeseci bila je -1,72 mL/min/1,73 m
c.T1147C or c.C1149G or c.C1149A
c.T1147C ili c.C1149G ili c.C1149A
In the ERT-experienced trial, renal function remained stable for up to 18 months of treatment with Galafold.
U ispitivanju s bolesnicima koji su prethodno primali ERT, funkcija bubrega ostala je stabilna do 18 mjeseci terapije lijekom Galafold.
Do not take this medicine if you are breast-feeding, until you have spoken with your doctor, pharmacist, or nurse.
Ne uzimajte lijek ako dojite prije nego što se savjetujete sa svojim liječnikom, ljekarnikom ili medicinskom sestrom.
It is not yet known whether this medicine passes into breast milk.
Nije poznato izlučuje li se ovaj lijek u majčino mlijeko.
Skin and subcutaneous tissue disorders
Poremećaji kože i potkožnog tkiva osip svrbež
1.478; n=34) in the Galafold group compared to -1.03 mL/min/1.73 m
(95 % CI: -2,272; 1,478; n = 34) u skupini koja je primala Galafold u usporedbi s -1,03 mL/min/1,73 m
The mutations not amenable to treatment with Galafold are listed in Table 3 below.
Mutacije koje isključuju odgovor na liječenje lijekom Galafold navedene su u tablici 3. u nastavku.
c.712A>C or c.714T>A or c.714T>G
c.712A>C ili c.714T>A ili c.714T>G
c.A924T or c.A924C
c.A924T ili c.A924C
c.A442C or c.T444A or c.T444G S148R
c.A442C ili c.T444A ili c.T444G S148R
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.
U ispitivanju s bolesnicima koji prethodno nisu primali ERT, analize ljestvice za ocjenu gastrointestinalnih simptoma pokazale su da je liječenje lijekom Galafold bilo povezano sa statistički značajnim (p < 0,05) poboljšanjima u odnosu na skupinu koja je primala placebo od početka ispitivanja do 6. mjeseca u području proljeva te refluksa za bolesnike koji su imali simptome na početku ispitivanja.
c.611G>A or 612G>A
c.611G>A ili 612G>A
Certain GLA mutations can result in the production of abnormally folded and unstable mutant forms of α-Gal A. Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of -Gal A, the genotypes of which are referred to as amenable mutations.
Migalastat je farmakološki šaperon koji je osmišljen kako bi se selektivno i reverzibilno te s visokim afinitetom vezao za aktivna mjesta određenih mutiranih oblika enzima -Gal A čiji genotipovi imaju mutacije koje omogućuju odgovor na liječenje.
c.442A>C or c.444T>A or c.444T>G
c.442A>C ili c.444T>A ili c.444T>G
The GLA mutations amenable and not amenable to treatment with Galafold are listed in Table 2 and Table 3 respectively below.
Mutacije gena GLA koje omogućuju i koje isključuju odgovor na liječenje lijekom Galafold navedene su u tablici 2., odnosno tablici 3. u nastavku.
UNKNOWN (del Exon3 and 4?)
NEPOZNATO (del Exon3 i 4?)
c.504A>C or c.504A>T
c.504A>C ili c.504A>T
c.1095T>A or c.1095T>G c.T1095A or c.T1095G
c.1095T>A ili c.1095T>G c.T1095A ili c.T1095G
c.C519A or c.C519G
c.C519A ili c.C519G
Protein sequence change
Promjena slijeda u proteinu
c.3G>A or c.3G>T or c.3G>C c.G3A or c.G3T or c.G3C
c.3G>A ili c.3G>T ili c.3G>C c.G3A ili c.G3T ili c.G3C
c.1045T>C or c.1045T>A c.T1045C or c.T1045A
c.1045T>C ili c.1045T>A c.T1045C ili c.T1045A
c.140G>A or 141G>A
c.140G>A ili 141G>A
• Painful stiff neck
• bolni ukočeni vrat (tortikolis)
Galafold is not indicated for use in patients with non-amenable mutations (see section 5.1).
Galafold nije indiciran za primjenu u bolesnika s mutacijama koje isključuju odgovor na liječenje (vidjeti dio 5.1).
c.1034C>G or c.1034C>A c.C1034G or c.C1034A
c.1034C>G ili c.1034C>A c.C1034G ili c.C1034A
c.216G>A or c.216G>T or c.216G>C
c.216G>A ili c.216G>T ili c.216G>C
c.G412A or c.G412C
c.G412A ili c.G412C
c.1023A >C or c.1023A>T
c.1023A >C ili c.1023A>T
c.C207A or c.C207G
c.C207A ili c.C207G
c.A124C or c.A124T
c.A124C ili c.A124T
c.C816A or c.C816G
c.C816A ili c.C816G
c.1046G>A or c.1047G>A c.G1046A or c.G1047A
c.1046G>A ili c.1047G>A c.G1046A ili c.G1047A
c.T139C or c.T139A
c.T139C ili c.T139A
c.1147T>C or c.1149C>G or c.1149C>A
c.1147T>C ili c.1149C>G ili c.1149C>A
c.1065C>A or c.1065C>G c.C1065A or c.C1065G
c.1065C>A ili c.1065C>G c.C1065A ili c.C1065G
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).
Srednja vrijednost promjene u odnosu na početnu vrijednost LVMi od 18. do 24. mjeseca u bolesnika s hipertrofijom lijevog ventrikula na početku (ženski bolesnici s početnom vrijednošću LVMi > 95 g/m ili muški bolesnici s početnom vrijednošću LVMi > 115 g/m2) iznosila je -18,6 (95 % CI: -38,2; 1,0; n = 8).
c.C159G or c.C159A
c.C159G ili c.C159A
c.G216A or c.G216T or c.G216C M72I
c.G216A ili c.G216T ili c.G216C M72I
c.G126A or c.G126C or c.G126T M42I
c.G126A ili c.G126C ili c.G126T M42I
c.G228C or c.G228A or c.G228T M76I
c.G228C ili c.G228A ili c.G228T M76I
c.148T>C or c.150C>G or c.150C>A
c.148T>C ili c.150C>G ili c.150C>A
c.G284A or c.G285A
c.G284A ili c.G285A
c.G103C or c.G103A
c.G103C ili c.G103A
c.T241C or c.T241A
c.T241C ili c.T241A
c.280T>A or c.281G>C
c.280T>A ili c.281G>C
c.561G>T or c.561G>A or c.561G>C
c.561G>T ili c.561G>A ili c.561G>C
c.C279G or c.C279A
c.C279G ili c.C279A
g.941_5845del p.?
g.941_5845del p.?(Exon1_2del)
c.924A>T or c.924A>C
c.924A>T ili c.924A>C
The effect of age was evaluated in a population pharmacokinetic analysis on plasma migalastat clearance in the ERT-naïve study population.
U analizi populacijske farmakokinetike ocijenjen je učinak dobi na klirens migalastata iz plazme u populaciji bolesnika koji nisu prethodno primali ERT.
Respiratory, thoracic, and mediastinal disorders
Poremećaji dišnog sustava, prsišta i sredoprsja dispneja epistaksa
c.G140A or G141A
c.G140A ili G141A
c.139T>C or c.139T>A
c.139T>C ili c.139T>A
c.T187A or c.G188C
c.T187A ili c.G188C
c.G982A or c.G982C
c.G982A ili c.G982C
Hepatic impairment No dosage adjustment of Galafold is required in patients with hepatic impairment (see section 5.2).
U bolesnika s oštećenjem jetre nije potrebna prilagodba doze lijeka Galafold (vidjeti dio 5.2).
c.412G>A or c.412G>C
c.412G>A ili c.412G>C
c.G261C or c.G261T
c.G261C ili c.G261T
Galafold is not recommended for use in patients with severe renal insufficiency, defined as estimated GFR less than 30 mL/min/1.73m
Ne preporučuje se primjena lijeka Galafold u bolesnika s teškom bubrežnom insuficijencijom, definiranom procijenjenom vrijednosti GFR-a manjom od 30 mL/min/1,73m
c.159C>G or c.159C>A
c.159C>G ili c.159C>A
c.G285T or c.G285C
c.G285T ili c.G285C
c.G540C or c.G540T
c.G540C ili c.G540T
c.153G>A or c.153G>T or c.153G>C
c.153G>A ili c.153G>T ili c.153G>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.
95,3 g/m za skupinu koja je primala Galafold i 92,9 g/m za skupinu koja je primala ERT, a srednja vrijednost promjene od početne vrijednosti u LVMi u 18. mjesecu bila je -6,6 (95 % Cl: -11,0; -2,1; n = 31) za Galafold i -2,0 (95 % Cl: -11,0; 7,0; n = 13) za ERT.
c.T881G or c.T881A
c.T881G ili c.T881A
c.G612T or c.G612C
c.G612T ili c.G612C
c.G131A or c.G132A
c.G131A ili c.G132A
c.228G>C or c.228G>A or c.228G>T
c.228G>C ili c.228G>A ili c.228G>T
c.817T>C or c.819T>A or c.819T>G
c.817T>C ili c.819T>A ili c.819T>G
c.337T>C or c.339T>A or c.339T>G
c.337T>C ili c.339T>A ili c.339T>G
c.G870A or c.G870C or c.G870T M290I
c.G870A ili c.G870C ili c.G870T M290I
c.G471C or c.G471T
c.G471C ili c.G471T
c.284G>A or c.285G>A
c.284G>A ili c.285G>A
c.C525G or c.C525A
c.C525G ili c.C525A
c.T166A or c.G167C
c.T166A ili c.G167C
No reduction in proteinuria was observed in patients treated with Galafold.
U bolesnika liječenih lijekom Galafold nije uočeno smanjenje proteinurije.
c.T552A or c.T552G
c.T552A ili c.T552G
c.G242A or c.G243A
c.G242A ili c.G243A
c.261G>C or c.261G>T
c.261G>C ili c.261G>T
c.G734A or c.G735A
c.G734A ili c.G735A
c.241T>C or c.241T>A
c.241T>C ili c.241T>A
c.187T>A or c.188G>C
c.187T>A ili c.188G>C
c.612G>T or c.612G>C
c.612G>T ili c.612G>C
Psychiatric disorders
Psihijatrijski poremećaji depresija
c.C233G or c.C233A
c.C233G ili c.C233A
c.124A>C or c.124A>T
c.124A>C ili c.124A>T
c.C305G or c.C305A
c.C305G ili c.C305A
c.552T>A or c.552T>G
c.552T>A ili c.552T>G
c.G132T or c.G132C
c.G132T ili c.G132C
c.G1074T or c.G1074C
c.G1074T ili c.G1074C
c.G243T or c.G243C
c.G243T ili c.G243C
c.242G>A or c.243G>A
c.242G>A ili c.243G>A
Headache and dizziness were the most common adverse reactions reported at doses of Galafold of up to 1250 mg and 2000 mg, respectively.
Najčešća nuspojava zabilježena pri dozama lijeka Galafold od najviše 1250 mg bila je glavobolja, odnosno pri dozama od najviše 2000 mg omaglica.
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.
Ako je prošlo više od 12 sati, trebate nastaviti uzimati Galafold onog dana i u ono doba za koje je predviđena sljedeća doza u skladu s rasporedom doziranja svakog drugog dana.
c.G1081A or c.G1081C
c.G1081A ili c.G1081C
c.G888A or c.G888T or c.G888C M296I
c.G888A ili c.G888T ili c.G888C M296I
UNKNOWN (del Exon3?)
NEPOZNATO (del Exon3?)
c.T258G or c.T258A
c.T258G ili c.T258A
c.471G>C or c.471G>T
c.471G>C ili c.471G>T
c.285G>T or c.285G>C
c.285G>T ili c.285G>C
c.C747G or c.C747A
c.C747G ili c.C747A
c.279C>G or c.279C>A
c.279C>G ili c.279C>A
c.T960G or c.T960A
c.T960G ili c.T960A
c.207C>A or c.207C>G
c.207C>A ili c.207C>G
c.982G>A or c.982G>C
c.982G>A ili c.982G>C
c.519C>A or c.519C>G
c.519C>A ili c.519C>G
c.C453G or c.C453A
c.C453G ili c.C453A
c.G609C or c.G609T
c.G609C ili c.G609T
Galafold is not recommended for use in patients with Fabry disease who have estimated GFR less than 30 mL/min/1.73 m
Ne preporučuje se primjena lijeka Galafold u bolesnika s Fabryjevom bolešću kod kojih je procijenjena vrijednost GFR-a manja od 30 mL/min/1,73 m
c.A840T or c.A840C
c.A840T ili c.A840C
c.131G>A or c.132G>A
c.131G>A ili c.132G>A
c.233C>G or c.233C>A
c.233C>G ili c.233C>A
c.T280A or c.G281C
c.T280A ili c.G281C
c.G567C or c.G567T
c.G567C ili c.G567T
c.243G>T or c.243G>C
c.243G>T ili c.243G>C
c.G144T or c.G144C
c.G144T ili c.G144C
• Stomach ache
• suha usta
c.T610C or c.T610A
c.T610C ili c.T610A
c.T514A or c.G515C
c.T514A ili c.G515C
c.1081G>A or c.1081G>C
c.1081G>A ili c.1081G>C
c.1074G>T or c.1074G>C
c.1074G>T ili c.1074G>C
c.816C>A or c.816C>G
c.816C>A ili c.816C>G
c.G288A or c.G288T or c.G288C M96I
c.G288A ili c.G288T ili c.G288C M96I
c.258T>G or c.258T>A
c.258T>G ili c.258T>A
c.525C>G or c.525C>A
c.525C>G ili c.525C>A
c.T859C or c.T859A
c.T859C ili c.T859A
c.A588T or c.A588C
c.A588T ili c.A588C
c.C897G or c.C897A
c.C897G ili c.C897A
Lot
Louth
c.540G>C or c.540G>T
c.540G>C ili c.540G>T
c.132G>T or c.132G>C
c.132G>T ili c.132G>C
UNKNOWN (del Exon1_2?)
NEPOZNATO (del Exon1_2?)
c.T408A or c.T408G
c.T408A ili c.T408G
c.T154A or c.G155C
c.T154A ili c.G155C
c.166T>A or c.167G>C
c.166T>A ili c.167G>C
UNKNOWN (del Exon5_7?)
NEPOZNATO (del Exon5_7?)
c.G831T or c.G831C
c.G831T ili c.G831C
c.609G>C or c.609G>T
c.609G>C ili c.609G>T
c.103G>C or c.103G>A
c.103G>C ili c.103G>A
c.T369G or c.T369A
c.T369G ili c.T369A
c.960T>G or c.960T>A
c.960T>G ili c.960T>A
c.G141C or c.G141T
c.G141C ili c.G141T
c.881T>G or c.881T>A
c.881T>G ili c.881T>A
c.G830A or c.G831A
c.G830A ili c.G831A
c.707G>A or c.708G>A
c.707G>A ili c.708G>A
Paraesthesia Dizziness Hypoaesthesia
Poremećaji živčanog sustava glavobolja parestezija omaglica hipoestezija
c.305C>G or c.305C>A
c.305C>G ili c.305C>A
c.144G>T or c.144G>C
c.144G>T ili c.144G>C
c.G860A or c.G861A
c.G860A ili c.G861A
c.141G>C or c.141G>T
c.141G>C ili c.141G>T
c.T465A or c.T465G
c.T465A ili c.T465G
c.453C>G or c.453C>A
c.453C>G ili c.453C>A
c.108G>C or c.108G>T
c.108G>C ili c.108G>T
c.610T>C or c.610T>A
c.610T>C ili c.610T>A
c.567G>C or c.567G>T
c.567G>C ili c.567G>T
c.G485A or c.G486A
c.G485A ili c.G486A
Dyspnoea Epistaxis
Prijavljivanje nuspojava
c.C966A or c.C966G
c.C966A ili c.C966G
c.C456A or c.C456G
c.C456A ili c.C456G
c.830G>A or c.831G>A
c.830G>A ili c.831G>A
c.G71A or c.G72A
c.G71A ili c.G72A
c.888G>A or c.888G>T or c.888G>C
c.888G>A ili c.888G>T ili c.888G>C
• Indigestion (dyspepsia)
• trnci u udovima (parestezija)
c.T676C or c.T676A
c.T676C ili c.T676A
c.734G>A or c.735G>A
c.734G>A ili c.735G>A
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).
Nakon praćenja u otvorenom nastavku ispitivanja, srednja vrijednost promjene u odnosu na početnu vrijednost LVMi u 36. mjesecu iznosila je -8,3 (95 % CI: -17,1; 0,4; n = 25), a u 48. mjesecu iznosila je -9,1 (95 % CI: -20,3; 2,0; n = 18).
c.870G>A or c.870G>C or c.870G>T
c.870G>A ili c.870G>C ili c.870G>T
c.G108C or c.G108T
c.G108C ili c.G108T
c.G439A or c.G439C
c.G439A ili c.G439C
c.G861C or c.G861T
c.G861C ili c.G861T
c.859T>C or c.859T>A
c.859T>C ili c.859T>A
c.860G>A or c.861G>A
c.860G>A ili c.861G>A
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
Nakon praćenja u otvorenom nastavku ispitivanju, srednja vrijednost promjene u odnosu na početnu vrijednost LVMi u bolesnika s hipertrofijom lijevog ventrikula na početku u 36. mjesecu iznosila je -30,0 (95 % CI: -57,9; -2,2; n = 4), a u 48. mjesecu iznosila je -33,1 (95 % CI: -60,9; -5,4; n = 4).
c.831G>T or c.831G>C
c.831G>T ili c.831G>C
c.G394A or c.G394C
c.G394A ili c.G394C
c.T648A or c.T648G
c.T648A ili c.T648G
c.485G>A or c.486G>A
c.485G>A ili c.486G>A
c.439G>A or c.439G>C
c.439G>A ili c.439G>C
c.966C>A or c.966C>G
c.966C>A ili c.966C>G
c.514T>A or c.515G>C
c.514T>A ili c.515G>C
c.G486C or c.G486T
c.G486C ili c.G486T
c.897C>G or c.897C>A
c.897C>G ili c.897C>A
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
Galafold nije ispitivan u bolesnika s Fabryjevom bolešću koji imaju GFR manji od 30 ml/min/1,73 m U ispitivanju koje je uključivalo davanje jedne doze Galafolda u ispitanika koji nisu imali Fabryjevu bolest s različitim stupnjevima bubrežne insuficijencije, izloženosti su se povećale 4,3 puta u ispitanika s teškim oštećenjem bubrega (GFR < 30 ml/min/1,73 m
c.465T>A or c.465T>G
c.465T>A ili c.465T>G
c.861G>C or c.861G>T
c.861G>C ili c.861G>T
c.A868C or c.A868T
c.A868C ili c.A868T
c.394G>A or c.394G>C
c.394G>A ili c.394G>C
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
Nakon prosječnog razdoblja od 3,4 godine liječenja, srednja godišnja stopa promjene u eGFRCKD-EPI iznosila je -0,74 mL/min/1,73 m2 (95 % CI: -1,89; 0,40; n = 41).
c.T667A or c.G668C
c.T667A ili c.G668C
c.588A>T or c.588A>C
c.588A>T ili c.588A>C
c.408T>A or c.408T>G
c.408T>A ili c.408T>G
c.154T>A or c.155G>C
c.154T>A ili c.155G>C
c.C666A or c.C666G
c.C666A ili c.C666G
c.71G>A or c.72G>A
c.71G>A ili c.72G>A
c.T834G or c.T834A
c.T834G ili c.T834A
c.102T>G or c.102T>A
c.102T>G ili c.102T>A
c.T70C or c.T70A
c.T70C ili c.T70A
c.T102G or c.T102A
c.T102G ili c.T102A
c.288G>A or c.288G>T or c.288G>C
c.288G>A ili c.288G>T ili c.288G>C
c.T894G or c.T894A
c.T894G ili c.T894A
c.G837C or c.G837T
c.G837C ili c.G837T
c.708G>C or c.708G>T
c.708G>C ili c.708G>T
c.837G>C or c.837G>T
c.837G>C ili c.837G>T
g.?_?dup c.?_?dup
Promjena slijeda u proteinu g.?_?dup c.?_?dup
c.G963C or c.G963T
c.G963C ili c.G963T
c.784T>A or c.784T>C
c.784T>A ili c.784T>C
c.T687A or c.T687G
c.T687A ili c.T687G
c.676T>C or c.676T>A
c.676T>C ili c.676T>A
c.T402G or c.T402A
c.T402G ili c.T402A