natgillin/translations-raw huggingface dataset:
If you take more Galafold than you should
Ako uzmete više lijeka Galafold nego što ste trebali
Take one capsule every other day at the same time of the day.
Uzmite jednu kapsulu lijeka Galafold svaki drugi dan u isto vrijeme.
• raised levels of creatine phosphokinase in blood tests
• povećane razine kreatin fosfokinaze u krvnim pretragama
Following ascending single oral doses (25-675 mg migalastat hydrochloride), no trends were found for clearance, CL/F).
Nakon povećavanja jednokratnih oralnih doza (25 - 675 mg migalastatklorida) nije primijećen nikakav trend za klirens CL/F.
If your condition worsens, your doctor may evaluate you further or may discontinue your treatment with Galafold.
Ako se Vaše stanje pogorša, Vaš liječnik može obaviti dodatnu procjenu ili prekinuti liječenje lijekom Galafold.
Plasma migalastat exposure (AUC0-∞) and Cmax demonstrated dose-proportional increases at migalastat hydrochloride oral doses from 50 mg to 1,250 mg.
Izloženost migalastatu u plazmi (AUC0-∞) i Cmax pokazali su povećanje proporcionalno dozi pri oralnim dozama migalastatklorida od 50 mg do 1250 mg.
Swallow the capsule whole.
Progutajte cijelu kapsulu.
Pharmacotherapeutic group: Various alimentary tract and metabolism products ATC code: A16AX14
Farmakoterapijska skupina: Različiti lijekovi s djelovanjem na probavni sustav i metabolizam ATK oznaka: A16AX14
Black iron oxide Potassium hydroxide
Tinta za označavanje šelak crni željezov oksid kalijev hidroksid
Non-clinical studies suggest no specific hazard for humans on the basis of single-and repeat-dose studies, with the exception of transient and fully reversible infertility in male rats associated with migalastat treatment.
Neklinička ispitivanja ne ukazuju na poseban rizik za ljude na temelju ispitivanja jednokratne doze i ponovljenih doza, uz iznimku prolazne i potpuno reverzibilne neplodnosti u mužjaka štakora povezane s liječenjem migalastatom.
UNKNOWN IVS6-10G>A; IVS6-22C>T c.1000-10G>A/1000-22C>T UNKNOWN
NEPOZNATO IVS6-10G>A; IVS6-22C>T c.1000-10G>A/1000-22C>T NEPOZNATO
In case of meaningful clinical deterioration, further clinical evaluation or discontinuation of treatment with Galafold should be considered.
U slučaju značajnog kliničkog pogoršanja potrebno je razmotriti treba li provesti dodatnu kliničku procjenu ili prekinuti liječenje lijekom Galafold.
In a rat 104-week carcinogenicity study, there was an increased incidence of pancreatic islet cell adenomas in males at a dose level 19-fold higher than the exposure (AUC) at the clinically efficacious dose.
U ispitivanju kancerogenosti u štakora u trajanju od 104 tjedna bila je povećana incidencija adenoma stanica otočića gušterače u mužjaka pri razinama doza 19 puta višima od izloženosti (AUC) pri klinički djelotvornoj dozi.
Limited data suggest that co-administration of a single dose of Galafold and a standard enzyme replacement therapy infusion results in an increased exposure to agalsidase of up to 5-fold.
Ograničeni podaci ukazuju na to da istodobna primjena jedne doze lijeka Galafold i infuzije standardne enzimske nadomjesne terapije dovodi do povećanja izloženosti agalzidazi do 5 puta.
Then, punch out the left-most capsule labelled Day 1.
Zatim izvadite prvu kapsulu s lijeve strane koja je označena natpisom 1.dan.
Complete reversibility was seen after 4 weeks off-dose.
Potpuna reverzibilnost zabilježena je nakon 4 tjedna od prestanka uzimanja lijeka.
All patients received the recommended dosage of 123 mg Galafold every other day.
Svi bolesnici primili su preporučenu dozu lijeka Galafold od 123 mg svaki drugi dan.
Women of childbearing potential/Contraception in males and females Galafold is not recommended in women of childbearing potential not using contraception.
Žene u reproduktivnoj dobi/kontracepcija u muškaraca i žena Galafold se ne preporučuje ženama u reproduktivnoj dobi koje ne koriste kontracepciju.
Fabry disease is caused by the lack of or a faulty enzyme called alpha-galactosidase A (α-Gal A).
Fabryjevu bolest uzrokuje nedostatak ili oštećenje enzima naziva alfa-galaktozidaza A (α-Gal A).
On your first day of taking this medicine from a new blister sleeve, record the date on the blister sleeve.
Prvog dana uzimanja lijeka iz nove blister kartice zabilježite datum na kartici.
Transient and fully reversible infertility in male rats was associated with migalastat treatment at all doses assessed.
Prolazna i potpuno reverzibilna neplodnost u mužjaka štakora bila je povezana s liječenjem migalastatom pri primjeni svih ispitivanih doza.
c.369+3_639+954del3129insT UNKNOWN (del Exon3 and 4?)
c.369+3_639+954del3129insT NEPOZNATO (del Exon3 i 4?)
If a double mutation is present on the same chromosome (males and females), that patient is amenable if the double mutation is present in one entry in Table 2 (e.g., D55V/Q57L).
Ako je dvostruka mutacija prisutna na istom kromosomu (i u muškaraca i u žena), takav će bolesnik odgovoriti na liječenje ako je dvostruka mutacija prisutna u jednom od unosa u tablici 2 (npr. D55V/Q57L).
UNKNOWN (delExon3_3'UTR?)
NEPOZNATO (delExon3_3'UTR?)
Similar findings have been noted pre-clinically following treatment with other iminosugars.
Slični nalazi primijećeni su pretklinički nakon liječenja drugim iminošećerima.
The first OLE trial (AT1001-041) included patients from Phase 2 and Phase 3 studies and has completed.
Prvi otvoreni nastavak ispitivanja (AT1001-041) uključivao je bolesnike iz ispitivanja faze 2 i faze 3 i završen je.
The effects of Galafold on fertility in humans have not been studied.
Učinci lijeka Galafold na plodnost u ljudi nisu ispitani.
Migalastat is not a substrate for P-glycoprotein (P-gP) in vitro and it is considered unlikely that migalastat would be subject to drug-drug interactions with cytochrome P450s.
Migalastat nije supstrat za P-glikoprotein (P-gP) in vitro i ne smatra se vjerojatnim da bi migalastat bio podložan interakciji lijekova s citokromima P450.
In the rabbit embryo-foetal toxicity study, findings including embryo-foetal death, a reduction in mean foetal weight, retarded ossification, and slightly increased incidences of minor skeletal abnormalities were observed only at doses associated with maternal toxicity.
U ispitivanju embrio-fetalne toksičnosti u kunića, nalazi koji uključuju embrio-fetalnu smrt, smanjenje srednje fetalne težine, usporenu osifikaciju i blago povećanu incidenciju malih koštanih abnormalnosti primijećeni su samo u dozama povezanima s toksičnošću za majku.
Weight increased
• porast težine
• are allergic to migalastat or any of the other ingredients of this medicine (listed in section 6) Warnings and precautions
• ako ste alergični na migalastat ili neki drugi sastojak ovog lijeka (naveden u dijelu 6.).
In healthy volunteers, the volume of distribution (Vz/F) of migalastat following ascending single oral doses (25-675 mg migalastat HCl) ranged from 77 to 133 L, indicating it is well distributed into tissues and greater than total body water (42 litres).
U zdravih dobrovoljaca volumen distribucije (Vz/F) migalastata nakon povećavanja jednokratne oralne doze (25 - 675 mg migalastat HCl) kretao se u rasponu od 77 do 133 l, što je pokazalo dobru distribuciju lijeka u tkiva, te prelazi ukupnu količinu vode u tijelu (42 litre).
You should not take Galafold if you are also receiving enzyme replacement therapy.
Ne smijete uzimati Galafold ako također primate enzimsku nadomjesnu terapiju.
Migalastat administered with a high-fat meal, or 1 hour before a high-fat or light meal, or 1 hour after a light meal, resulted in significant reductions of 37% to 42% in mean total migalastat exposure (AUC0-∞) and reductions of 15% to 40% in mean peak migalastat exposure (Cmax) compared with the fasting state.
Migalastat primijenjen uz obrok s visokim udjelom masti ili sat vremena prije obroka s visokim udjelom masti ili laganog obroka ili sat vremena nakon laganog obroka doveo je do značajnih smanjenja srednje ukupne izloženosti migalastatu (AUC0-∞) od 37 % do 42 %, te do smanjenja srednje maksimalne izloženosti migalastatu (Cmax) od 15 do 40 % u usporedbi s primjenom natašte.
c.207_369+651del814ins231 UNKNOWN (del Exon2?)
c.207_369+651del814ins231 NEPOZNATO (del Exon2?)
In the absence of similar findings in females, no findings in the genotoxicity battery or in the carcinogenicity study with Tg.rasH2 mice, and no pre-neoplastic pancreatic findings in the rodents or monkeys, this observation in male rats is not considered related to treatment and its relevance to humans is unknown.
U nedostatku sličnih nalaza u ženki, rezultata baterija ispitivanja genotoksičnosti ili ispitivanja kancerogenosti s miševima Tg.rasH2 te nedostatku preneoplastičnih nalaza u gušterači u glodavaca ili majmuna, navedena pojava u mužjaka štakora ne smatra se povezanom s liječenjem te njezin značaj za ljude nije poznat.
In addition, migalastat is not a substrate for MATE1, MATE2-K, OAT1, OAT3, or OCT2, nor is it an inhibitor of OATP1B1, OATP1B3, OAT1, OAT3, OCT1, OCT2, MATE1, or MATE2-K human uptake transporters.
Osim toga, migalastat nije supstrat za MATE1, MATE2-K, OAT1, OAT3 ili OCT2 niti je inhibitor humanih transportera za unos OATP1B1, OATP1B3, OAT1, OAT3, OCT1, OCT2, MATE1 ili MATE2-K.
Furthermore, migalastat is not an inhibitor or a substrate of CYP1A2, 2A6, 2B6, 2C8, 2C9, 2C19, 2D6, 2E1, or 3A4/5.
Nadalje, migalastat nije inhibitor niti supstrat enzima CYP1A2, 2A6, 2B6, 2C8, 2C9, 2C19, 2D6, 2E1 ili 3A4/5.
If you forget to take Galafold
Ako ste zaboravili uzeti Galafold
In the ERT-experienced trial, an analysis of a composite clinical outcome composed of renal, cardiac, and cerebrovascular events, or death, showed that the frequency of events observed in the Galafold treatment group was 29% compared to 44% in the ERT group over 18 months.
U ispitivanju s bolesnicima koji su prethodno primali ERT, analiza kompozitnog kliničkog ishoda koji se sastojao od bubrežnih, srčanih i cerebrovaskularnih događaja ili smrti, je pokazala kako je učestalost događaja primijećenih u skupini koja je primala Galafold iznosila 29 %, u usporedbi s 44 % u skupini koja je primala ERT tijekom 18 mjeseci.
Galafold is to be taken every other day.
Galafold je potrebno uzimati svaki drugi dan.
Migalastat is not a substrate for MDR1 or BCRP, nor is it an inhibitor of BCRP, MDR1, or BSEP human efflux transporters.
Migalastat nije supstrat za MDR1 ili BCRP, niti je inhibitor humanih efluksnih transportera BCRP, MDR1 ili BSEP.
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%.
Apsolutna bioraspoloživost (AUC) za jednokratnu oralnu dozu migalastatklorida od 150 mg ili jednokratnu intravensku infuziju od 150 mg u trajanju od 2 sata iznosila je otprilike 75 %.
Capsule contents Pregelatinised starch (maize) Magnesium stearate
Prethodno geliran škrob (kukuruzni) Magnezijev stearat
Following a single oral dose of 150 mg migalastat hydrochloride solution, the time to peak plasma concentration was approximately 3 hours.
Nakon jednokratne oralne doze otopine migalastatklorida od 150 mg, vrijeme do postizanja maksimalnih koncentracija u plazmi iznosilo je otprilike 3 sata.
UNKNOWN (Exon2_4dup?)
NEPOZNATO (Exon2_4dup?)
Based upon in vivo data, migalastat is a substrate for UGT, being a minor elimination pathway.
Na temelju podataka in vivo zaključuje se da je migalastat supstrat za UGT enzim koji predstavlja manje značajan put eliminacije.
Elderly population No dosage adjustment is required based on age (see section 5.2).
Starija populacija Nije potrebna prilagodba doze na temelju dobi (vidjeti dio 5.2).
This enzyme defect leads to abnormal deposits of a fatty substance known as globotriaosylceramide (GL-3) in kidneys, heart, and other organs, leading to the symptoms of Fabry disease.
Oštećenje tog enzima dovodi do abnormalnog taloženja masne tvari poznate pod nazivom globotriaozilceramid (GL-3) u bubrezima, srcu i ostalim organima, što dovodi do pojave simptoma Fabryjeve bolesti.
Alternate daily between taking the capsule on odd numbered days and punching out the perforated white circles on even numbered days, up to and including day 28.
Uzimajte kapsule na neparne dane i bušite perforirane krugove na parne dane do (uključujući) 28. dana.
Do not eat food at least 2 hours before and 2 hours after taking your medicine to give a minimum 4 hours fast.
Nemojte jesti najmanje 2 sata prije i 2 sata poslije uzimanja lijeka kako biste omogućili najmanje 4 sata posta.
Migalastat binding stabilizes these mutant forms of -Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes.
Vezivanje migalastata stabilizira te mutirane oblike enzima -Gal A u endoplazmatskom retikulumu i olakšava njihov pravilan transport do lizosoma.
There are limited data from the use of Galafold in pregnant women.
Postoje ograničeni podaci o primjeni lijeka Galafold u trudnica.
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.
Kvalitativna smanjenja razina GL-3 primijećena su u više vrsta bubrežnih stanica: podocitima, mezangijalnim stanicama, glomerularnim endotelnim stanicama, tijekom 12 mjeseci liječenja lijekom Galafold.
Pack size of 14 capsules.
Pakiranje od 14 kapsula.
Migalastat did not affect fertility in female rats.
Migalastat nije imao učinka na plodnost u ženki štakora.
If a dose is missed entirely for the day, the patient should take the missed dose of Galafold only if it is within 12 hours of the normal time the dose is taken.
Ako je propustio dozu za cijeli jedan dan, bolesnik treba uzeti propuštenu dozu lijeka Galafold samo ako je to unutar 12 sati od uobičajenog vremena uzimanja doze.
(95% CI: -3.636, 1.575; n=18) in the ERT group.
(95 % CI: -3,636; 1,575; n = 18) u skupini koja je primala ERT.
Fax: +353 (0) 1 588 6851 e-mail: info@amicusrx.co.uk
Fax: +353 (0) 1 588 6851 e-pošta: info@amicusrx.co.uk
Plasma lyso- Gb3 levels also remained low in patients on ERT for up to 18 months.
Razine lyso-Gb3 u plazmi također su ostale niske u bolesnika koji su primali ERT u razdoblju do 18 mjeseci.
The infertility associated with migalastat treatment was reported at clinically relevant exposures.
Neplodnost povezana s liječenjem migalastatom zabilježena je pri klinički značajnim izloženostima.
In rabbits, developmental toxicity was observed only at maternally toxic doses (see section 5.3).
U kunića je razvojna toksičnost uočena samo pri dozama toksičnim za majku (vidjeti dio 5.3).
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.
Mutacije u genu GLA koje uzrokuju Fabryjevu bolest dovode do nedostatka lizosomskog enzima -galaktozidaze A (-Gal A) koji je potreban za metabolizam supstrata glikosfingolipida (npr. supstrata GL-3, lyso-Gb3).
c.760-762delGTT or c.761-763del c.760_762delGTT or c.761_763del p.V254del
c.760-762delGTT ili c.761-763del c.760_762delGTT ili c.761_763del p.V254del
Reduced -Gal A activity is, therefore, associated with the progressive accumulation of substrate in vulnerable organs and tissues, which leads to the morbidity and mortality associated with Fabry disease.
Stoga se smanjena aktivnost enzima -Gal A povezuje s progresivnim nakupljanjem supstrata u podložnim organima i tkivima što dovodi do morbiditeta i mortaliteta povezanih s Fabryjevom bolešću.
Based upon in vitro data, migalastat is not an inducer of CYP1A2, 2B6, or 3A4.
Na temelju in vitro podataka, utvrđeno je da migalastat nije induktor enzima CYP1A2, 2B6 ili 3A4.
This is a common spontaneous tumour in ad libitum-fed male rats.
Riječ je o čestom spontanom tumoru u mužjaka štakora hranjenih ad libitum.
No clinically significant differences were observed during the initial 6-month placebo-controlled period.
Nisu zabilježene klinički značajne razlike u pogledu LVMi tijekom početnog 6-mjesečnog, placebom kontroliranog razdoblja.
What Galafold contains - The active substance is migalastat.
Što sadrži Galafold - Djelatna tvar je migalastat.
After Day 2, continue moving right on the blister sleeve.
Nakon 2.dana nastavite uzimati kapsule na blister kartici u desnu stranu.
Similar findings have been noted pre-clinically following treatment with other iminosugars (see section 5.3).
Slični nalazi primijećeni su u pretkliničkoj fazi nakon liječenja drugim iminošećerima (vidjeti dio 5.3).
To access the package leaflet, scan the code below.
Za pristup uputi o lijeku očitajte sljedeći kod.
Table 1: Adverse reactions with Galafold in clinical trials System Organ Class
Tablica 1.: Nuspojave lijeka Galafold zabilježene u kliničkim ispitivanjima Klasifikacija organskih sustava
UNKNOWN (E66_Y123del; del Exon2)
NEPOZNATO (E66_Y123del; del Exon2)
You should only take Galafold once every other day.
Galafold smijete uzimati samo jedanput svaki drugi dan.
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
- Drugi sastojci su: Sadržaj kapsule: prethodno geliran kukuruzni škrob i magnezijev stearat Ovojnica kapsule: želatina, titanijev dioksid i indigotin Tinta za označavanje: šelak, crni željezov oksid i kalijev hidroksid
Table 3: Mutations not amenable to Galafold (migalastat)
Tablica 3.: Mutacije koje isključuju odgovili na lijek Galafold (migalastat)
Galafold is not intended for concomitant use with enzyme replacement therapy (see section 4.4).
Galafold nije namijenjen za istodobnu primjenu s enzimskom nadomjesnom terapijom (vidjeti dio 4.4).
Take Galafold capsule every other day and punch out the perforated circle on the blister sleeve on the days you are not taking Galafold.
Kapsulu lijeka Galafold uzmite svaki drugi dan te probušite perforirani krug na blister kartici na one dane kada ne uzimate Galafold.
Elderly (> 65 years) Clinical studies of Galafold included small number of patients aged 65 and over.
Klinička ispitivanja lijeka Galafold obuhvatila su mali broj bolesnika u dobi od 65 godina i starijih.
The clinical efficacy and safety of Galafold have been evaluated in two Phase 3 pivotal trials and two open-label extension (OLE) trials.
Klinička djelotvornost i sigurnost lijeka Galafold ocijenjene su u dvama pivotalnim ispitivanjima faze 3 te u dva otvorena nastavka ispitivanja.
Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1).
Galafold je indiciran za dugotrajno liječenje odraslih i adolescenata u dobi od 16 godina i starijih s potvrđenom dijagnozom Fabryjeve bolesti (nedostatak α-galaktozidaze A) i koji imaju mutacije koje omogućuju odgovor na liječenje (vidjeti tablice u dijelu 5.1).
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.
Ako je prošlo više od 12 sati, bolesnik treba nastaviti uzimati Galafold onog dana i u ono doba za koje je predviđena sljedeća doza u skladu s rasporedom doziranja svakog drugog dana.
It is not yet known if this medicine affects fertility in men.
Još nije poznato utječe li ovaj lijek na plodnost u muškaraca.
UNKNOWN (E66_Y123del; del Exon2?)
NEPOZNATO (E66_Y123del; del Exon2?)
MINIMUM PARTICULARS TO APPEAR ON INTERMEDIATE PACKAGING
PODACI KOJE MORA NAJMANJE SADRŽAVATI UNUTARNJE PAKIRANJE
If you stop taking Galafold Do not stop taking this medicine without talking to your doctor.
Nemojte prestati uzimati lijek bez prethodnog razgovora sa svojim liječnikom.
During the first period (18-months) ERT-experienced patients were randomised to switch from ERT to Galafold or continue with ERT.
Tijekom prvog razdoblja (18-mjesečno) bolesnici koji su prethodno primali ERT bili su randomizirani na prelazak s ERT-a na lijek Galafold ili na nastavak uzimanja ERT-a.
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).
Srednja vrijednost duljine izloženosti dozi lijeka Galafold od 123 mg (koja se nalazi na tržištu) svakog drugog dana u bolesnika koji su završili ispitivanje AT1001-041 bila je 3,57 (±1,23) godina (n = 85).
Marketing Authorisation Holder Amicus Therapeutics Europe Limited Block 1, Blanchardstown Corporate Park Ballycoolen Road
Nositelj odobrenja za stavljanje lijeka u promet Amicus Therapeutics Europe Limited Block 1, Blanchardstown Corporate Park Ballycoolen Road
• Muscle pain (myalgia)
• bol u mišićima (mialgija)
Each capsule contains migalastat hydrochloride equivalent to 123 mg migalastat.
Svaka kapsula sadrži migalastatklorid koji odgovara 123 mg migalastata.
See package leaflet for further instructions.
Vidjeti uputu o lijeku za dodatne upute.
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).
Prvo ispitivanje faze 3 (ATTRACT) bilo je randomizirano otvoreno ispitivanje s aktivnim usporednim lijekom kojim su ocijenjene djelotvornost i sigurnost lijeka Galafold u usporedbi s enzimskom nadomjesnom terapijom (ERT, engl. enzyme replacement therapy) (agalzidaza beta, agalzidaza alfa) u 52 muških i ženskih bolesnika s Fabryjevom bolešću koji su primali ERT prije pridruživanja ispitivanju te koji imaju mutacije koje omogućuju odgovor na liječenje (ispitivanje s bolesnicima koji su prethodno primali ERT).
Size 2 hard capsule (6.4x18.0 mm) with an opaque blue cap and opaque white body with “A1001” printed in black, containing white to pale brown powder.
Tvrda kapsula veličine 2 (6,4 x 18,0 mm) s neprozirnom plavom kapicom i neprozirnim bijelim tijelom s oznakom „A1001” ispisanom crnom bojom koja sadrži prašak bijele do svijetlosmeđe boje.
How to take Galafold
Kako uzimati Galafold
While taking Galafold you should use effective birth control.
Tijekom uzimanja lijeka Galafold trebate koristiti učinkovitu kontracepciju.
Fabry disease is a progressive X-linked lysosomal storage disorder which affects males and females.
Fabryjeva bolest je progresivni X-vezani poremećaj nakupljanja u lizosomima koji se javlja u muškaraca i žena.
In the ERT-experienced trial, following 18 months of treatment with Galafold there was a statistically significant decrease in LVMi (p< 0.05).
U ispitivanju s bolesnicima koji su prethodno primali ERT, a nakon 18-mjesečnog liječenja lijekom Galafold, došlo je do statistički značajnog smanjenja LVMi (p < 0,05).
Once in lysosomes dissociation of migalastat restores -Gal A activity, leading to the catabolism of GL-3 and related substrates.
Jednom kada je u lizosomima, odvajanje migalastata obnavlja aktivnost enzima -Gal A, što dovodi do katabolizma supstrata GL-3 i srodnih supstrata.
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
Drugi otvoreni nastavak ispitivanja (AT1001-042) uključuje i bolesnike koji su prešli iz nastavka ispitivanja AT1001-041 i one koji su prešli izravno iz ispitivanja faze 3, ATTRACT, i još uvijek je u tijeku.
BLISTER FOIL
FOLIJA BLISTERA
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).
Bolesnici koji su bili randomizirani u skupinu koja je primala placebo u 1. fazi te koji su prešli na Galafold u 6. mjesecu (2.faza) također su pokazali statistički značajno smanjenje inkluzija GL-3 u intersticijskim kapilarama u 12. mjesecu (-0,33 ± 0,15; -58 %) (p = 0,014).