natgillin/translations-raw huggingface dataset:
Replagal is used to treat adult patients, as well as adolescents and children from the age of 7, with confirmed diagnosis of Fabry Disease.
Zdravilo Replagal je namenjeno zdravljenju odraslih bolnikov ter mladostnikov in otrok, starih 7 let ali več, s potrjeno diagnozo Fabryjeve bolezni.
Infusion related reactions have been experienced by 9 patients of the naïve population (31.0%) compared to 27 patients of the switched population (38.0%).
Z infuzijo povezane reakcije so se pojavile pri 9 predhodno nezdravljenih bolnikih (31,0 %) v primerjavi s 27 bolniki, ki so prešli na zdravilo Replagal (38,0 %).
A review of cardiac events showed that infusion reactions may be associated with hemodynamic stress triggering cardiac events in patients with pre-existing cardiac manifestations of Fabry disease.
Pregled srčnih dogodkov je pokazal, da so lahko reakcije na infundiranje povezane s hemodinamičnim stresom, ki sproži srčne dogodke pri bolnikih z obstoječimi srčnimi manifestacijami
Agalsidase alfa has been produced in a human cell line to provide for a human glycosylation profile that can influence uptake by mannose-6-phosphate receptors on the surface of target cells.
Agalzidazo alfa so proizvedli v humani celični liniji zaradi priprave na humani profil glikozilacije, ki lahko vpliva na privzem preko manoza-6 fosfatnih receptorjev na površini ciljnih celic.
Patients received the recommended dosage of 0.2 mg/kg of Replagal.
Bolniki so dobili priporočeni odmerek 0,2 mg/kg zdravila Replagal.
No increase in the incidence of adverse events was detected for this patient.
Pri tem bolniku ni bilo opaziti nobenega povečanja incidence neželenih učinkov.
The metabolic effects were also associated with clinically significant weight gain, increased sweating and increased energy.
Presnovni učinki so bili povezani tudi s klinično pomembnim povečanjem telesne mase, močnejšim potenjem in povečano količino energije.
There is very limited data on pregnancies exposed to Replagal.
Podatki o nosečnicah, izpostavljenih zdravilu Replagal so zelo omejeni.
Compared with placebo, treatment with Replagal also reduced accumulation of Gb3.
V primerjavi s placebom je zdravljenje z zdravilom Replagal zmanjšalo tudi kopičenje Gb3.
Common: may affect up to 1 in 10 people: - tingling or numbness or pain in fingers or toes, change in the taste of food, eyes tearing, blink reflex abnormal, ears ringing, shakes, prolonged sleep - palpitations, increased heart rate, increased blood pressure - cough, chest pain or tightness, hoarseness, sore or tight throat, sticky throat secretions, runny nose, cold symptoms - vomiting, abdominal pain or discomfort, diarrhoea - acne, red or itchy or mottled skin, rash at the infusion site - back or limb pain, muscle pain, joint pain, muscle and bone discomfort, swelling of the extremities or joints - feeling cold or hot, flu-like symptoms, feeling sick, feeling lack of energy
- ščemenje ali otrplost ali bolečina v prstih na rokah ali nogah, spremembe okusa hrane, solzenje oči, nenormalen refleks mežikanja, zvenenje v ušesih, tresenje, podaljšano spanje - palpitacije, zvišan srčni utrip, zvišan krvni tlak - kašelj, bolečina ali stiskanje v prsnem košu, hripavost, bolečina ali stiskanje v grlu, vlažen izloček iz grla, izcedek iz nosu, simptomi prehlada - bruhanje, bolečina ali neprijeten občutek v trebuhu, driska - akne, rdeča, srbeča ali lisasta koža, izpuščaj na mestu infundiranja - bolečina v hrbtu ali okončinah, bolečina v mišicah, bolečina v sklepih, neprijeten občutek v mišicah in kosteh, otekanje okončin ali sklepov - občutek vročine ali mraza, simptomi, podobni gripi, slabo počutje, pomanjkanje energije
*agalsidase alfa is the human protein α-galactosidase A produced in a human cell line by genetic engineering technology.
* agalzidaza alfa je humani protein α-galaktozidaza A, izdelan s tehnologijo genskega inženiringa v humani celični liniji.
After 12 to 54 months of therapy, 17% of Replagal treated patients were still antibody positive whereas 7% showed evidence for the development of immunologic tolerance, based on the disappearance of IgG antibodies over time.
Po 12 do 54 mesecih zdravljenja je bilo 17% bolnikov, ki so jih zdravili z zdravilom Replagal, še vedno pozitivnih na protitelesa, pri 7 % pa so se kazali znaki imunske tolerance, kar se je kazalo kot izginjanje protiteles IgG s časom.
Elimination half-lives were 108 ± 17 minutes in males compared to 89 ± 28 minutes in females and volume of distribution was approximately 17% body weight in both sexes.
108 ± 17 minut pri bolnikih v primerjavi z 89 ± 28 minut pri bolnicah, porazdelitveni volumen pa je bil približno 17 % telesne mase pri obeh spolih.
Patients switched from agalsidase beta to Replagal had a safety profile consistent with that observed in other clinical experience.
Varnostni profil pri bolnikih, ki so prešli z algazidaze beta na zdravilo Replagal, je bil skladen z opaženim v drugih kliničnih izkušnjah.
Agalsidase alfa catalyses the hydrolysis of Gb3, cleaving a terminal galactose residue from the molecule.
Agalzidaza alfa katalizira hidrolizo Gb3 z odcepitvijo terminalnega ostanka galaktoze iz molekule.
The enzyme is then removed from the cells and made into a sterile concentrate for solution for infusion.
Encim se nato odstrani iz celic in predela v sterilni koncentrat za pripravo raztopine za infundiranje.
Other infusion-related symptoms may include dizziness and hyperhidrosis.
Med drugimi simptomi, povezanimi z infundiranjem, sta lahko omotica in hiperhidroza.
Since no preservative is present, it is recommended that administration is started as soon as possible.
Ker raztopina ne vsebuje konzervansov, je priporočljivo, da začnete z dajanjem čim prej.
As metabolism is expected to occur by peptide hydrolysis, impaired liver function is not expected to affect the pharmacokinetics of agalsidase alfa in a clinically significant manner.
Ker je pričakovati, da bo presnova potekala s hidrolizo peptidov, ni verjetno, da bi jetrna okvara klinično pomembno vplivalo na farmakokinetiko agalzidaze alfa.
Based on the similarity of pharmacokinetic properties of agalsidase alfa in both males and females, tissue distribution in major tissues and organs is also expected to be comparable in male and female patients.
Zaradi podobnosti farmakokinetičnih lastnosti agalzidaze alfa pri bolnicah in bolnikih je pričakovati, da je tudi porazdelitev encima po poglavitnih tkivih in organih podobna pri bolnicah in bolnikih.
Data with patients indicates that at least 0.1mg/kg is required to achieve a pharmacodynamics response.
Podatki, pridobljeni pri bolnikih kažejo, da je za farmakodinamični odziv potreben vsaj odmerek 0,1 mg/kg.
Kidney biopsy specimens revealed a significant increase in the fraction of normal glomeruli and a significant decrease in the fraction of glomeruli with mesangial widening in patients treated with Replagal in contrast to the patients treated with placebo.
so vzorci biopsije ledvic pokazali pomembno povečanje deleža normalnih glomerulov in pomembno zmanjšanje deleža glomerulov z mezangialno razširitvijo v primerjavi z bolniki, ki so prejemali placebo.
The presence of extensive renal damage (eGFR <60mL/min) may limit the renal response to enzyme replacement therapy.
Hude poškodbe ledvic (eGFR [receptor za epidermalni rastni faktor] < 60 ml/min) lahko omejijo odziv ledvic na encimsko nadomestno zdravljenje.
The selection of 0.2 mg/kg dose (infused over 40 minutes) for the registration clinical studies was intended to temporarily saturate the ability of the mannose-6phosphate receptors to internalize the agalsidase alfa in the liver and allow distribution of enzyme to other relevant organ tissues.
Izbira odmerka 0,2 mg/kg (40-minutna infuzija) za klinične študije za pridobitev dovoljenja za promet z zdravilom je bila namenjena začasnemu nasičenju sposobnosti manoza-6-fosfatnih receptorjev za internalizacijo agalzidaze alfa v jetrih in omogočanju porazdelitve encima v druga pomembna organska tkiva.
In male paediatric Fabry patients ≥ 7 years of age, hyperfiltration can be the earliest manifestation of renal involvement in the disease.
Pri moških pediatričnih bolnikih ≥ 7 let s Fabryjevo boleznijo je lahko hiperfiltracija najzgodnejši znak težav z ledvicami.
Uncommon: may affect up to 1 in 100 people: - severe allergic (anaphylactic-type) reaction
Občasni (pojavijo se lahko pri največ 1 od 100 bolnikov): - huda alergijska reakcija (anafilaktičnega tipa).
Instructions for use, handling and disposal
Navodila za uporabo in ravnanje z zdravilom ter odstranjevanje
Adverse drug reactions reported in patients with history of end stage renal disease were similar to those reported in the general patient population.
Neželeni učinki, o katerih so poročali pri bolnikih z anamnezo ledvične bolezni v zadnji fazi, so bili podobni tistim, o katerih so poročali pri splošni populaciji bolnikov.
Renal elimination of agalsidase alfa is considered to be a minor clearance pathway since pharmacokinetic parameters are not altered by impaired renal function.
Ledvično izločanje agalzidaze alfa velja za manj pomembno pot izločanja, saj ledvična okvara ne vpliva na spremembo farmakokinetičnih parametrov.
Chemical and physical in use stability has been demonstrated for 24 hours at 25°C.
Vendar pa je razredčena raztopina dokazano kemično in fizikalno stabilna 24 ur pri temperaturi 25 °C.
Do not use Replagal if you notice that there is discolouration or other foreign particles present.
Ne uporabljajte zdravila Replagal če opazite, da je razbarvan ali če vsebuje tuje delce.
It is not known whether Replagal crosses the placenta.
Ni znano, ali zdravilo Replagal prehaja skozi placento.
• Since no preservative is present, it is recommended that administration is started as soon as possible after dilution.
• Ker raztopina ne vsebuje konzervansov je priporočljivo, da začnete z dajanjem čim prej po redčenju.
No studies have been conducted with respect to parturition or peri/post-natal development.
Študije vpliva na kotenje oziroma peri in postnatalni razvoj niso bile opravljene.
Administer the infusion solution over a period of 40 minutes using an intravenous line with an integral filter.
Raztopino infundirajte 40 minut z uporabo intravenskega kanala z vgrajenim filtrom.
The overall incidence of SAEs by treatment group did not show any obvious effect of treatment regimen on the SAE profile of the different treatment groups.
Skupna incidenca hudih neželenih učinkov v zdravljenih skupinah ni pokazala, da bi režim zdravljenja kakor koli očitno vplival na profil hudih neželenih učinkov v skupinah z različnimi zdravljenji.
Table 1 lists adverse reactions reported for the 177 patients treated with Replagal in clinical trials, including 21 patients with history of end stage renal disease, 24 paediatric patients (7 to 17 years of age) and 17 female patients, and from post-marketing spontaneous reports.
Seznam neželenih reakcij v obliki preglednice V preglednici 1 so navedene neželene reakcije, o katerih so poročali pri 177 bolnikih, ki so jih v kliničnih preskušanjih zdravili z zdravilom Replagal; med njimi je bilo 21 bolnikov z anamnezo ledvične bolezni v končni fazi, 24 pediatričnih bolnikov (7-17 let) in 17 žensk, ter neželene reakcije, o katerih so spontano poročali v obdobju trženja.
Side effects reported in children were, in general, similar to those reported in adults.
Neželeni učinki, o katerih so poročali pri otrocih, so bili v splošnem podobni tistim pri odraslih.
Treatment with the enzyme has been shown to reduce accumulation of Gb3 in many cell types including endothelial and parenchymal cells.
da zdravljenje s tem encimom zmanjša kopičenje Gb3 v različnih vrstah celic, vključno z endotelijskimi in parenhimalnimi celicami.
Agalsidase alfa is a form of the human enzyme α-galactosidase.
Agalzidaza alfa je oblika človeškega encima α-galaktozidaze.
Consistent with the clinical effects of Replagal, treatment with the enzyme reduced accumulation of Gb3 in many cell types, including renal glomerular and tubular epithelial cells, renal capillary endothelial cells (cardiac and dermal capillary endothelial cells were not examined) and cardiac myocytes.
Skladno s kliničnimi učinki zdravila Replagal je encimsko zdravljenje zmanjšalo kopičenje Gb3 v številnih vrstah celic, vključno z ledvičnimi glomerulnimi in tubulnimi epitelijskimi celicami, celicami kapilarnega endotelija ledvic (celic kapilarnega endotelija srca in kože niso pregledali) in srčnimi mišičnimi celicami.
Most undesirable effects were mild to moderate in severity.
Neželeni učinki so bili večinoma blagi do zmerni.
What Replagal looks like and contents of the pack
Izgled zdravila Replagal in vsebina pakiranja
Two patients with right bundle branch block in the studies conducted reverted to normal following therapy with Replagal.
Pri dveh bolnikih z blokado desne veje srčnega prevodnega sistema, ki sta sodelovala v opravljenih študijah, se je stanje po zdravljenju z zdravilom Replagal normaliziralo.
Metabolism and nutrition disorders peripheral oedema
Presnovne in prehranske motnje periferni edem
Replagal treatment should be supervised by a physician experienced in the management of patients with Fabry Disease or other inherited metabolic diseases.
Zdravljenje z zdravilom Replagal mora nadzorovati zdravnik, ki ima izkušnje z obravnavo bolnikov s Fabryjevo boleznijo ali drugimi dednimi presnovnimi boleznimi.
In clinical studies, neuropathic pain medicinal products (such as carbamazepine, phenytoin and gabapentin) were administered concurrently to most patients without any evidence of interaction.
V kliničnih študijah ob sočasni uporabi zdravila Replagal in zdravil proti nevropatični bolečini (kot so karbamazepin, fenitoin in gabapentin) pri večini bolnikov ni bilo znakov interakcij.
It is produced by switching on the gene for α-galactosidase A in cells.
Pridobiva se z vklopom gena za α-galaktozidazo v celicah.
However, infusion related reactions (pyrexia, dyspnoea, chest pain) and pain exacerbation occurred more frequently.
Vendar pa so bile reakcije, povezane z infundiranjem (pireksija, dispneja, bolečina v prsnem košu), in eksacerbacija bolečine pogostejše.
If mild or moderate acute infusion reactions occur, medical attention must be sought immediately and appropriate actions instituted.
Če se pojavijo blage ali zmerne akutne infuzijske reakcije, mora bolnik takoj poiskati medicinsko pomoč in je treba uvesti ustrezne ukrepe.
After 12 to 18 months of maintenance therapy, Replagal improved renal function as measured by inulin based glomerular filtration rate by 8.7 ± 3.7 ml/min.
Po 12 do 18 mesecih vzdrževalnega zdravljenja je zdravilo Replagal izboljšalo delovanje ledvic, merjeno s stopnjo glomerulne filtracije, temelječe na inulinu, za 8,7 ± 3,7 ml/min (p = 0,030).
Adverse drug reactions reported in the paediatric population (children and adolescents) were, in general, similar to those reported in adults.
Neželeni učinki zdravila, o katerih so poročali pri pediatričnih bolnikih (otrocih in mladostnikih), so bili na splošno podobni neželenim učinkom, o katerih so poročali pri odraslih.
Replagal has no or negligible influence on the ability to drive and use machines.
4.7 Vpliv na sposobnost vožnje in upravljanja s stroji Zdravilo Replagal nima vpliva ali ima zanemarljiv vpliv na sposobnost vožnje in upravljanja s stroji.
Once diluted, the solution should be mixed gently but not shaken.
Po redčenju morate raztopino nežno premešati, vendar je ne smete stresati.
Twelve to 18 months of treatment with Replagal resulted in improvement in quality of life (QoL), as measured by validated instruments.
12 do 18 mesecev zdravljenja z zdravilom Replagal je izboljšalo kakovost življenja, izmerjeno s preverjenimi metodami.
As α-galactosidase A is itself an enzyme, it would be an unlikely candidate for cytochrome P450 mediated drug-drug interactions.
Ker je sama α-galaktozidaza A encim, je malo verjetno, da bi lahko prihajalo do interakcij z drugimi zdravili s pomočjo posredovanja citokroma P450.
The recommended dose is an infusion of 0.2 mg for every kg you weigh.
Priporočeni odmerek je infuzija 0,2 mg na vsak kilogram telesne mase.
Treatment with Replagal resulted in an 11.5 g decrease in left ventricular mass as measured by magnetic resonance imaging (MRI) in the controlled study, while patients receiving placebo exhibited an increase in left ventricular mass of 21.8 g. In addition, in the first study involving 25 patients, Replagal effected a significant reduction in cardiac mass after 12 to 18 months of maintenance therapy (p<0.001).
V kontrolirani študiji je zdravljenje z zdravilom Replagal povzročilo zmanjšanje mase levega prekata za 11,5 g (izmerjeno z magnetnoresonančnim slikanjem (MRI), pri bolnikih, ki so prejemali placebo, pa je prišlo do povečanja mase levega prekata za 21,8 g. Tudi v prvi študiji, v katero je bilo vključenih 25 bolnikov, je zdravilo Replagal po 12 do 18 mesecih vzdrževalnega zdravljenja povzročilo pomembno zmanjšanje srčne mase (p < 0,001).
Infusion related reactions involving the heart including heart rhythm problems, heart muscle ischemia and heart failure, may occur in patients with Fabry disease involving the heart structures (frequency “not known” (cannot be estimated from the available data)).
Pri bolnikih s Fabryjevo boleznijo, ki zajema tudi strukture srca, se lahko pojavijo reakcije srca, povezane z infuzijo, vključno s težavami s srčnim ritmom, ishemijo srčne mišice in popuščanjem srca (pogostost »neznana« (ni mogoče oceniti iz razpoložljivih podatkov)).
The safety and efficacy of Replagal was assessed in two randomised, double blind, placebo controlled studies and open label extension studies, in a total of forty patients with a diagnosis of Fabry Disease based on clinical and biochemical evidence.
Varnost in učinkovitost zdravila Replagal so ovrednotili v dveh randomiziranih dvojno slepih, s placebom kontroliranih študijah in odprtih nadaljevalnih študijah, pri 40 bolnikih z diagnozo
In subsequent studies, in male paediatric patients above the age of 7, a reduction in pain was observed after 9 and 12 months of Replagal therapy compared to pretreatment baseline.
V nadaljnjih študijah pri moških pediatričnih bolnikih , starejših od 7 let, so zmanjšanje bolečine opazili po 9 in 12 mesecih zdravljenja z zdravilom Replagal, v primerjavi z izhodiščnimi vrednostmi.
3.5 ml of concentrate for solution for infusion in a 5 ml vial (Type I glass) with a stopper (fluoro-resin coated butyl rubber), a one piece seal (aluminium) and flip-off cap.
3,5 ml koncentrata za raztopino za infundiranje v 5 ml viali (steklo tipa 1) z zamaškom iz butilne gume s fluorovo smolnato prevleko in enodelno aluminijasto zaporko s ploščico.
MARKETING AUTHORISATION NUMBER(S) EU/1/01/189/001
ŠTEVILKA(E) DOVOLJENJA (DOVOLJENJ) ZA PROMET EU/1/01/189/001
This would be about 14 mg or 4 vials (glass bottles) of Replagal for an average size (70 kg) individual.
(70 kg) je to približno 14 mg ali 4 viale (stekleničke) zdravila Replagal.
Reproduction toxicity studies in female rats and rabbits have shown no effect on pregnancy or the developing foetus.
Študije vpliva na sposobnost razmnoževanja pri samicah podgan in kuncev niso pokazale nobenega vpliva na brejost in na razvijajoči se zarodek.
Limited data are available in patients on dialysis or post-kidney transplantation, no dose adjustment is recommended.
Pri bolnikih na dializi ali po presaditvi ledvic so na voljo omejeni podatki.
In children (aged 7-18 years), Replagal administered at 0.2 mg/kg was cleared faster from the circulation than in adults.
Pri otrocih, starih od 7 do 18 let, je bil očistek zdravila Replagal iz obtoka pri odmerku 0,2 mg/kg hitrejši kot pri odraslih.
Hypersensitivity reactions have been reported.
Poročali so o preobčutljivostnih reakcijah.
Serious infusion reactions have been reported uncommonly; symptoms reported include pyrexia, rigors, tachycardia, urticaria, nausea/vomiting, angioneurotic oedema with throat tightness, stridor and swollen tongue.
O resnih infuzijskih reakcijah so poročali občasno; med njihovimi simptomi so bili zvišanje telesne temperature, okorelost, tahikardija, urtikarija, navzea/bruhanje, angionevrotski edem s stiskanjem grla, stridor in otekanje jezika.
In male paediatric Fabry patients plasma Gb3 decreased 40-50% after 6 months of Replagal therapy 0.2 mg/kg and this reduction persisted after a total 4 years of treatment in 11 patients.
Pri moških pediatričnih bolnikih s Fabryjevo boleznijo se je po 6 mesecih zdravljenja z zdravilom Replagal v odmerku 0,2 mg/kg vrednost Gb3 v plazmi zmanjšala za 40-50 %; to zmanjšanje pa se je pri 11 bolnikih ohranilo tudi po 4 letih zdravljenja.
Longer term therapy (48-54 months) resulted in stabilisation of GFR in male patients with normal baseline GFR (≥ 90 mL/min/1.73 m2) and with mild to moderate renal dysfunction (GFR 60 to < 90 mL/min/1.73 m2), and in slowing of the rate of decline in renal function and progression to end-stage renal disease in male Fabry patients with more severe renal dysfunction (GFR 30 to < 60 mL/min/1.73 m2).
GFR (≥ 90 ml/min/1,73 m2) in z blago do zmerno ledvično okvaro (GFR 60 do < 90 ml/min/1,73 m2) povzročilo stabilizacijo vrednosti GFR, pri moških bolnikih s Fabryjevo boleznijo s hujšo ledvično okvaro (GFR 30 do < 60 ml/min/1,73 m2) pa upočasnilo slabšanje delovanja ledvic in napredovanje do končne faze ledvične bolezni.
Following a single intravenous dose of 0.2 mg/kg, agalsidase alfa had a biphasic distribution and elimination profile from the circulation.
Po enem intravenskem odmerku 0,2 mg/kg je bil profil porazdelitve in izločanja agalzidaze alfa iz krvnega obtoka dvofazen.
This pain reduction persisted through 4 years of Replagal therapy in 9 patients (in patients 7 - 18 years of age).
bolnikih trajalo 4 leta zdravljenja z zdravilom Replagal (pri bolnikih 7 - 18 let).
Vascular disorders flushing hypertension hypotension
Žilne bolezni pordelost hipertenzija hipotenzija
What you need to know before Replagal is given
Kaj morate vedeti, preden boste dobili zdravilo Replagal Zdravila Replagal ne smete dobiti
Eye disorders corneal reflex decreased, lacrimation increased
Očesne bolezni zmanjšan refleks roženice, povečano solzenje
Most side effects are mild to moderate.
Neželeni učinki so večinoma blagi do zmerni.
Based on limited data this percentage has been found to be lower (7%) in the male paediatric population.
Na podlagi navedenih podatkov je ta odstotek manjši
Replagal is administered at a dose of 0.2 mg/kg body weight every other week by intravenous infusion over 40 minutes.
Zdravilo Replagal se daje v odmerku 0,2 mg/kg telesne mase vsak drugi teden s 40-minutno intravensko infuzijo.
Infusion-related symptoms may also include dizziness, hyperhidrosis, hypotension, cough, vomiting and fatigue.
Simptomi, povezani z infundiranjem, lahko vključujejo tudi omotico, hiperhidrozo, hipotenzijo, kašelj, bruhanje in utrujenost.
Single doses ranging from 0.007 - 0.2 mg enzyme per kg body weight were administered to adult male patients as 20 - 40 minute intravenous infusions while female patients received 0.2 mg enzyme per kg body weight as 40 minute infusions.
Odrasli bolniki so prejeli posamezne odmerke v razponu od 0,007 do 0,2 mg encima na kilogram telesne mase v obliki 20 do 40-minutnih intravenskih infuzij, medtem ko so bolnice prejele 0,2 mg encima na kilogram telesne mase v obliki 40-minutnih intravenskih infuzij.
Tell your doctor if you use any medicines containing chloroquine, amiodarone, benoquin or gentamicin.
Če uporabljate katero koli zdravilo, ki vsebuje klorokin, amiodaron, benokvin ali gentamicin, morate o tem obvestiti zdravnika.
Hypersensitivity, including anaphylaxis, has been reported.
Poročali so o preobčutljivosti, vključno z anafilaksijo.
After the first 6 months of therapy mean decreases of approximately 20 - 50 % were observed in plasma, urine sediment, liver, kidney, and heart biopsy samples.
Po prvih 6 mesecih zdravljenja so opažali srednje zmanjšanje kopičenja Gb3 v plazmi, sedimentu seča ter vzorcih biopsije jeter, ledvic in srca za približno 20 do 50%.