natgillin/translations-raw huggingface dataset:
When compared to the measurements at 6 months, disease control was unchanged after 18 and 24 months of miglustat monotherapy (20 and 6 patients, respectively). 
U usporedbi s mjerenjima provedenim nakon 6 mjeseci, kontrola bolesti bila je nepromijenjena nakon 18 i 24 mjeseca monoterapije miglustatom (20 odnosno 6 bolesnika).
PHARMACEUTICAL FORM AND CONTENTS 84 x 1 hard capsules 
OBLIK I SADRŽAJ 84 x 1 tvrda kapsula
Data in severe renal impairment are limited to two patients with creatinine clearance in the range 18 - 29 ml/min and cannot be extrapolated below this range. 
Podatci kod teškog poremećaja funkcije bubrega ograničeni su na dva bolesnika s klirensom kreatinina u rasponu od 18 - 29 ml/min i ne mogu se ekstrapolirati ispod tog raspona.
The most abundant metabolite in urine was miglustat glucuronide accounting for 5% of the dose. 
Najprisutniji metabolit u urinu bio je miglustat glukuronid koji je činio 5% doze.
MINIMUM PARTICULARS TO APPEAR ON BLISTERS BLISTER 
PODACI KOJE MORA NAJMANJE SADRŽAVATI BLISTER BLISTER
This study also indicated that miglustat has no or limited effect on the pharmacokinetics of imiglucerase. 
To ispitivanje ukazuje također na to da miglustat nema nikakvo ili ima ograničeno djelovanje na farmakokinetiku imigluceraze.
These data suggest a decrease in CL/F by at least 70% in patients with severe renal impairment. 
Ti podatci ukazuju na smanjenje CL/F za najmanje 70% u bolesnika s teškim poremećajem funkcije bubrega.
Depression, insomnia, libido decreased Nervous system disorders Very common 
Depresija, nesanica, smanjen libido Poremećaji živčanog sustava Vrlo često
BATCH NUMBER<, DONATION AND PRODUCT CODES> Lot 
13. BROJ SERIJE<, OZNAKE DONACIJE I PROIZVODA> Serija
Your doctor will perform some tests before and during treatment with Yargesa to assess this (see section 2). 
Liječnik će provesti neke pretrage prije i za vrijeme liječenja lijekom Yargesa kako bi to procijenio (vidjeti dio 2).
In type 1 Gaucher disease, these tremors were described as an exaggerated physiological tremor of the hands. 
Kod Gaucherove bolesti tipa 1, ti su tremori opisani kao pretjerani fiziološki tremor ruku.
Changes in lactation were observed in female rats in a 1-year study. 
Tijekom 1-godišnjeg ispitivanja uočene su promjene u laktaciji kod ženskih štakora.
If you do get any of these effects, please seek medical advice from your doctor as soon as possible. 
Ako imate bilo koju od tih nuspojava, zatražite savjet svog liječnika što je prije moguće.
In addition 12 paediatric patients were enrolled in an uncontrolled substudy for an overall average duration of 3.1 years and up to 4.4 years. 
Osim toga 12 pedijatrijskih bolesnika bilo je uključeno u nekontrolirano podispitivanje u ukupnom prosječnom trajanju od 3,1 godine pa do 4,4 godine.
Other medicines and Yargesa Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines. 
Obavijestite svog liječnika ili ljekarnika ako uzimate, nedavno ste uzeli ili biste mogli uzeti bilo koje druge lijekove.
Both data sets included paediatric, adolescent and adult patients with an age range of 1 year to 43 years. 
Oba seta podataka uključuju pedijatrijske, adolescentne i odrasle bolesnike dobnog raspona od 1 do 43 godine.
Driving and using machines Yargesa may make you feel dizzy. 
Upravljanje vozilima i strojevima Yargesa može uzrokovati omaglicu.
Data to support safety and efficacy of Yargesa in Niemann-Pick type C disease come from a prospective open-label clinical trial and a retrospective survey. 
Podatci koji podupiru sigurnost i djelotvornost Yargesa u Niemann-Pickovoj bolesti tipa C potječu iz prospektivnog otvorenog kliničkog ispitivanja i retrospektivne analize.
Niemann-Pick type C disease is a very rare, invariably progressive and eventually fatal neurodegenerative disorder characterised by impaired intracellular lipid trafficking. 
Niemann-Pickova bolest tipa C je vrlo rijedak, uvijek progresivan i u konačnici smrtonosan neurodegenerativni poremećaj koji karakterizira poremećen intracelularni transport lipida.
The main effects common to all species were weight loss and diarrhoea, and, at higher doses, damage to the gastrointestinal mucosa (erosions and ulceration). 
Glavni učinci zajednički svim vrsta bili su gubitak tjelesne težine i proljev, a pri višim dozama, oštećenje gastrointestinalne sluznice (erozije i ulceracije).
Of these patients, 132 had type 1 Gaucher disease, and 40 had Niemann-Pick type C disease. 
Od tih bolesnika, 132 su imala Gaucherovu bolest tipa 1, a 40 je imalo Niemann-Pickovu bolest tipa
If you have Niemann-Pick type C disease, fats such as glycosphingolipids build up in the cells of your brain. 
Ako imate Niemann-Pickovu bolest tipa C, masti kao što su glikosfingolipidi nakupljaju se u stanicama Vašeg mozga.
Limited data suggest that co-administration of miglustat and enzyme replacement with imiglucerase in patients with type 1 Gaucher disease may result in decreased exposure to miglustat (approximate reductions of 22% in Cmax and 14% in AUC were observed in a small parallel-group study). 
Ograničeni podaci ukazuju na to da istodobna primjena miglustata i enzimskog nadomjestka s imiglucerazom u bolesnika s Gaucherovom bolesti tipa 1 može za posljedicu imati smanjenu izloženost miglustatu (približno smanjenje od 22% u Cmax i 14% u AUC zabilježeno u malom ispitivanju s paralelnim skupinama).
For children less than 12 years old, your doctor will adjust the dose for Niemann-Pick type C disease. 
Kod djece mlađe od 12 godina, liječnik će prilagoditi dozu za Niemann-Pickovu bolest tipa C.
Contraceptive measures should be used by women of childbearing potential. 
Metode kontracepcije trebaju koristiti i žene reproduktivne dobi.
An open-label, non-comparative, 2-year study enrolled 42 patients with type 1 Gaucher disease, who had received a minimum of 3 years of ERT and who fulfilled criteria of stable disease for at least 2 years. 
Nekomparativno, 2-godišnje otvoreno ispitivanje uključivalo je 42 bolesnika s Gaucherovom bolesti tipa 1, koji su najmanje 3 godine primali ERT, a koji su zadovoljavali kriterij stabilne bolesti od najmanje 2 godine.
After 3 years of continuous miglustat treatment, mean reductions in liver and spleen organ volume were 17.5% and 29.6%, respectively. 
Nakon 3 godine neprekidnog liječenja miglustatom, prosječno smanjenje volumena jetre i slezene bilo je 17,5% odnosno 29,6%.
There was no drug-related increase in tumour incidence in male or female rats in any other organ. 
Nije bilo povećanja incidencije tumora povezanih s lijekom u muških ili ženskih štakora ni u kojem drugom organu.
The metabolite accounting for this has not been identified, but may accumulate and reach concentrations exceeding those of miglustat at steady state. 
Metabolit koji je za to odgovoran nije utvrđen, ali se može akumulirati i dosegnuti koncentracije koje nadilaze one miglustata pri stanju dinamičke ravnoteže.
This means a daily maximum of three capsules (300 mg). 
To znači da je dnevni maksimum tri kapsule (300 mg).
Yargesa is a white hard capsule that consists of an opaque white cap and body with “708” printed in black on the body. 
Yargesa je bijela tvrda kapsula s neprozirnom bijelom kapicom i tijelom na kojem je crnom bojom otisnuto „708“.
In a pooled analysis of uncontrolled data, bone mineral density Z-scores at the lumbar spine and femoral neck increased by more than 0.1 units from baseline in 27 (57%) and 28 (65%) of the patients with longitudinal bone density measurements. 
U objedinjenoj analizi nekontroliranih podataka su Z-vrijednosti mineralne gustoće kosti lumbalne kralježnice i vrata bedrene kosti povećane za više od 0,1 jedinice od početka u 27 (57%) i 28 (65%) bolesnika s longitudinalnim mjerenjima gustoće kostiju.
The clinical trial included 29 adult and juvenile patients in a 12-month controlled period, followed by extension therapy for an average total duration of 3.9 years and up to 5.6 years. 
U kliničko ispitivanje bilo je uključeno 29 odraslih i mlađih bolesnika u 12-mjesečnom kontroliranom razdoblju, nakon čega je slijedilo produljeno liječenje u prosječnom ukupnom trajanju od 3,9 godina pa do 5,6 godina.
Sometimes your doctor will need to reduce the dose or stop Yargesa treatment to stop the tremor. 
Ponekad će Vam liječnik reći da smanjite dozu ili potpuno prekinete liječenje lijekom Yargesa kako bi otklonio tremor.
Yargesa contains the active substance miglustat which belongs to a group of medicines that affect metabolism. 
Yargesa sadrži djelatnu tvar miglustat koja pripada skupini lijekova koji djeluju na metabolizam.
This can result in liver and spleen enlargement, changes in the blood and bone disease. 
To za posljedicu može imati povećanje jetre i slezene, promjene u krvi i bolest kostiju.
Niemann-Pick type C disease 
Niemann-Pickova bolest tipa C
At steady state, the concentration of miglustat in cerebrospinal fluid of six type 3 Gaucher disease patients was 31.4-67.2% of that in plasma. 
U stanju dinamičke ravnoteže, koncentracija miglustata u cerebrospinalnom likvoru kod bolesnika s 6 Gaucherovom bolesti tipa 3 bila je 31,4-67,2% koncentracije u plazmi.
The efficacy of Yargesa in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established. 
Djelotvornost lijeka Yargesa kod djece i adolescenata u dobi od 0 do 17 godina s Gaucherovom bolesti tipa 1 nije ustanovljena.
The survey included a case series of 66 patients treated with Yargesa outside of the clinical trial for a mean duration of 1.5 years. 
Analiza je uključivala seriju slučajeva od 66 bolesnika liječenih Yargesa izvan kliničkog ispitivanja u prosječnom trajanju od 1,5 godine.
In addition, inhibitory action on a non-lysosomal glycosylceramidase has been demonstrated experimentally in vitro. 
Osim toga, inhibitorno djelovanje na nelizosomalne glikozilceramidaze eksperimentalno se pokazalo in vitro.
No patient showed rapid deterioration of type 1 Gaucher disease following the switch to miglustat monotherapy. 
Niti jedan bolesnik nije pokazao brzo pogoršanje Gaucherove bolesti tipa 1 nakon prelaska na monoterapiju miglustatom.
Capsule shell gelatin purified water titanium dioxide (E171) 
Ovojnica kapsule želatina, pročišćena voda, titanijev dioksid (E171).
Twenty-eight patients with mild to moderate type 1 Gaucher disease were enrolled in this 12-month non-comparative study, and 22 patients completed the study. 
Dvadeset i osam bolesnika s blagom do umjerenom Gaucherovom bolesti tipa 1 uključeno je u ovo 12-mjesečno nekomparativno ispitivanje, a 22 bolesnika završila su ispitivanje.
This medicine does not require any special storage conditions. 
Lijek ne zahtijeva posebne uvjete čuvanja.
The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. 
Uobičajeno liječenje Gaucherove bolesti tipa 1 je enzimsko nadomjesno liječenje.
The most common serious adverse reaction reported with miglustat treatment in clinical studies was peripheral neuropathy (see section 4.4). 
Najčešća ozbiljna nuspojava zabilježena liječenjem miglustatom u kliničkim ispitivanjima bila je periferna neuropatija (vidjeti dio 4.4).
Limited data in patients with Fabry disease and impaired renal function showed that CL/F decreases with decreasing renal function. 
Ograničeni podaci za bolesnike s Fabryjevom bolesti i poremećajem funkcije bubrega pokazuju da CL/F opada sa slabljenjem funkcije bubrega.
Gastrointestinal events, mainly diarrhoea, have been observed in more than 80% of patients, either at the outset of treatment or intermittently during treatment (see section 4.8). 
Kod više od 80% bolesnika primijećeni su gastrointestinalni događaji, uglavnom proljev, bilo u početku liječenja ili na mahove tijekom liječenja (vidjeti dio 4.8).
In some patients there were reductions in platelet count and increases in chitotriosidase activity indicating that miglustat monotherapy may not maintain the same control of disease activity in all patients. 
U nekih je bolesnika došlo do smanjenja broja trombocita i do povećanja aktivnosti hitotriozidaze što ukazuje na to da monoterapija miglustatom ne može u svih bolesnika jednako držati bolest pod nadzorom.
The neurological manifestations are considered secondary to the abnormal accumulation of glycosphingolipids in neuronal and glial cells. 
Neurološke manifestacije se smatraju sekundarnima na abnormalno nakupljanje glikosfingolipida u živčanim i glijalnim stanicama.
Of these, 18 patients at baseline were within established therapeutic goals for liver and spleen volume, haemoglobin levels, and platelet counts, and 16 patients remained within all these therapeutic goals at Month 24. 
Među njima je bilo 18 bolesnika s početnim volumenom jetre i slezene, razinama hemoglobina i brojem trombocita unutar utvrđenih terapijskih ciljeva, a 16 bolesnika je ostalo unutar svih tih terapijskih ciljeva u 24. mjesecu.
Further effects seen in animals at doses that result in exposure levels similar to or moderately higher than the clinical exposure level were: changes in lymphoid organs in all species tested, transaminase changes, vacuolation of thyroid and pancreas, cataracts, nephropathy and myocardial changes in rats. 
Nadalje učinci zabilježeni u životinja pri dozama koje su uzrokovale razine izloženosti slične ili umjereno više od kliničkih razina izloženosti, bili su: promjene na limfoidnim organima u svih ispitanih vrsta, promjene vrijednosti transaminaza, vakuolizacija štitnjače i gušterače, katarakta, nefropatije i promjene miokarda u štakora.
In type 1 Gaucher disease, a substance called glucosylceramide is not removed from your body. 
Kod Gaucherove bolesti tipa 1tvar nazvana glukozilceramid se ne uklanja iz Vašeg tijela.
Clinical benefit has been assessed at 24 and 36 months in 13 patients. 
Klinička korist ocijenjena je kod 13 bolesnika nakon 24 i 36 mjeseci.
Peripheral neuropathy seems to be more common in patients with type 1 Gaucher disease compared to the general population. 
Čini se da je periferna neuropatija češća u bolesnika s Gaucherovom bolesti tipa 1 u usporedbi s općom populacijom.
At 12 months, there was a mean reduction in liver organ volume of 12.1% and a mean reduction in spleen volume of 19.0%. 
U 12 mjeseci prosječno smanjenje volumena jetre bilo je za 12,1% i prosječno smanjenje volumena slezene bilo je za 19,0%.
Other studies revealed changes in sperm parameters (motility and morphology) consistent with an observed reduction in fertility. 
Ostala su ispitivanja otkrila promjene u parametrima sperme (motilitet i morfologija) sukladne opažanjima smanjene plodnosti.
There are no pharmacokinetic data available in patients with liver impairment, in children or adolescents with type 1 Gaucher disease or in the elderly (> 70 years). 
Nema dostupnih farmakokinetičkih podataka za bolesnike s oštećenjem funkcije jetre, djecu ili adolescente s Gaucherovom bolesti tipa 1 ili za starije bolesnike (> 70 godina).
This can result in disturbances in neurological functions such as slow eye movements, balance, swallowing, and memory, and in seizures. 
To za posljedicu može imati poremećaje neuroloških funkcija kao što su sporo pokretanje oka, ravnoteža, gutanje i pamćenje, te epileptički napadaji.
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness. 
U sklopu svake kategorije učestalosti, nuspojave su prikazane prema opadajućoj ozbiljnosti.
Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1). 
Yargesa se smije koristiti samo u liječenju bolesnika kod kojih enzimska nadomjesna terapija nije prikladna (vidjeti dijelove 4.4 i 5.1).
PVC and polychlorotrifluoroethylene (PCTFE) blister sealed with aluminium foil containing 21 capsules. 
Blister od PVC-a i poliklorotrifluoroetilena (PCTFE) zatvoren aluminijskom folijom koji sadrži 21 kapsulu.
- the other ingredients are sodium starch glycolate (type A), povidone (K-29/32), magnesium stearate, gelatin, purified water, titanium dioxide (E171), printing ink (shellac glaze, iron oxide black (E172), propylene glycol and concentrated ammonia solution) 
- drugi sastojci su: natrijev škroboglikolat (vrsta A), povidon (K-29/32), magnezijev stearat, želatina, pročišćena voda, titanijev dioksid (E171), tinta za označavanje (šelak glazura, crni željezov oksid (E172), propilenglikol i koncentrirana otopina amonijaka).
Eighteen patients then continued to receive miglustat under an optional extended treatment protocol. 
Osamnaest bolesnika je tada nastavilo primati miglustat u neobaveznom protokolu nastavka liječenja.
If your diarrhoea does not respond to these measures, or if you have any other abdominal complaint, consult your doctor. 
Ako proljev ne reagira na te mjere, ili ako osjećate bilo kakvu drugu bol u trbuhu, obratite se svom liječniku.
Dizziness has been reported as a common adverse reaction, and patients suffering from dizziness should not drive or use machines. 
Omaglica je zabilježena kao česta nuspojava pa bolesnici koji pate od omaglice ne bi trebali upravljati vozilom ili raditi sa strojevima.
Miglustat is mainly eliminated by renal excretion, with urinary recovery of unchanged drug accounting for 70-80% of the dose. 
Miglustat se uglavnom eliminira izlučivanjem bubrezima, pri čemu se u urinu pronašlo 70 - 80% doze neizmijenjenog lijeka.
Based on mg/kg/day and corrected for differences in faecal excretion, the doses corresponded to 8, 16 and 33/19 times the highest recommended human dose (200 mg t.i.d.). 
Doze koje se temelje na mg/kg/dan i korigirane za razlike pri izlučivanju fecesa, odgovaraju 8, 16 i 33/19 puta višim preporučenim dozama za ljude (200 mg triput dnevno).
The efficacy and safety of miglustat has not been specifically evaluated in patients with severe Gaucher disease. 
Djelotvornost i sigurnost miglustata nije posebno procjenjivana kod bolesnika s teškom Gaucherovom bolesti.
Tremor Approximately 37% of patients in clinical trials in type 1 Gaucher disease, and 58% of patients in a clinical trial in Niemann-Pick type C disease reported tremor on treatment. 
Približno 37% bolesnika u kliničkim ispitivanjima kod Gaucherove bolesti tipa 1 i 58% bolesnika u kliničkim ispitivanjima kod Niemann-Pickove bolesti tipa C izvijestilo je o tremoru tijekom liječenja.
Male patients should maintain reliable contraceptive methods while taking Yargesa (see sections 4.4 and 5.3). 
Muški bolesnici trebaju primjenjivati pouzdane metode kontracepcije dok uzimaju lijek Yargesa (vidjeti dijelove 4.4 i 5.3).
The most common adverse reactions reported in clinical studies with miglustat were diarrhoea, flatulence, abdominal pain, weight loss and tremor (see section 4.4). 
Najčešće nuspojave zabilježene u kliničkim ispitivanjima s miglustatom bile su proljev, flatulencija, bol u trbuhu, gubitak težine i tremor (vidjeti dio 4.4).
Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease (see sections 4.4, and 5.1). 
Yargesa je indicirana za liječenje progresivne neurološke manifestacije u odraslih bolesnika i pedijatrijskih bolesnika s Niemann-Pickovom bolesti tipa C (vidjeti dijelove 4.4 i 5.1).
In some patients, temporary dose reduction may be necessary. 
Kod nekih će bolesnika možda biti potrebno privremeno smanjenje doze.
In clinical practice, miglustat-induced gastrointestinal events have been observed to respond to individualised diet modification (for example reduction of sucrose, lactose and other carbohydrate intake), to taking miglustat between meals, and/or to anti-diarrhoeal medicinal products such as loperamide. 
U kliničkoj praksi, primijećeno je da gastrointestinalni događaji potaknuti miglustatom, reagiraju na individualiziranu prilagodbu prehrane (na primjer smanjeno uzimanje saharoze, laktoze i drugih ugljikohidrata), uzimanje miglustata između obroka, i/ili na lijekove protiv proljeva poput loperamida.
Studies in animals have shown reproductive toxicity, including dystocia (see section 5.3). 
Ispitivanja na životinjama pokazala su reproduktivnu toksičnost, uključujući i distociju (vidjeti dio 5.3).
A second open, controlled study of miglustat randomised 36 patients who had received a minimum of 2 years of treatment with ERT, into three treatment groups: continuation with imiglucerase, imiglucerase in combination with miglustat, or switch to miglustat. 
U drugom otvorenom, kontroliranom, randomiziranom ispitivanju na 36 bolesnika koji su najmanje 2 godine bili liječeni ERT-om, u tri skupine liječenja: nastavak liječenja imiglucerazom, imiglucerazom u kombinaciji s miglustatom, ili prelazak na miglustat.
Yargesa is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy. 
Yargesa se primjenjuje samo kada se bolesnik smatra nepodobnim za enzimsko nadomjesno liječenje.
Twenty-one patients completed 24 months of miglustat treatment. 
Dvadeset jedan bolesnik završio je liječenje miglustatom u trajanju od 24 mjeseca.
Tremor usually began within the first month, and in many cases resolved during treatment after between 1 and 3 months. 
Tremor obično počinje liječenja između 1. i 3. mjeseca.
While the numbers of subjects with mild and moderate renal impairment were very small, the data suggest an approximate decrease in CL/F of 40% and 60% respectively, in mild and moderate renal impairment (see section 4.2). 
Iako je broj ispitanika s blagim ili umjerenim poremećajem funkcije bubrega bio vrlo malen, podatci ukazuju na približno povećanje CL/F od 40% odnosno 60%, kod blagog ili umjerenog poremećaja funkcije bubrega (vidjeti dio 4.2).
All patients should undergo baseline and repeat neurological evaluation. 
Sve bolesnike treba neurološki procijeniti na početku i kasnije ponoviti neurološku procjenu.
There is a close relationship between renal function and clearance of miglustat, and exposure to miglustat is markedly increased in patients with severe renal impairment (see section 5.2). 
Postoji uska povezanost između funkcije bubrega i klirensa miglustata, a izloženost miglustatu je znatno povećana u bolesnika s teškim oštećenjem funkcije bubrega (vidjeti dio 5.2).
Miglustat has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease. 
Miglustat nije procjenjivan kod bolesnika s anamnezom značajne gastrointestinalne bolesti, uključujući i upalnu bolest crijeva.
Most serious side effects 
Najozbiljnije nuspojave
If you have diarrhoea, your doctor may ask you to change your diet to reduce your lactose and carbohydrate intake such as sucrose (cane sugar), or not to take Yargesa together with food, or to temporarily reduce your dose. 
Ako imate proljev liječnik može zatražiti prilagodbu prehrane kako biste smanjili uzimanje laktoze i drugih ugljikohidrata kao što je saharoza (šećer iz trske), ili neuzimanje lijeka Yargese s hranom ili privremeno smanjenje doze.
Pack size: 84 x 1 capsules. 
Veličina pakiranja: 84 x 1 kapsula.
They could be signs of peripheral neuropathy, due to side effects of Yargesa or they could be due to existing conditions. 
To mogu biti znakovi periferne neuropatije, koji se javljaju zbog nuspojava lijeka Yargesa ili su se javili zbog postojeće bolesti.
Pharmacokinetic data indicate increased systemic exposure to miglustat in patients with renal impairment. 
Farmakokinetički podatci ukazuju na povećanu sistemsku izloženost miglustatu u bolesnika s oštećenjem funkcije bubrega.
Miglustat has been studied in indications where certain events reported as adverse reactions, such as neurological and neuropsychological symptoms/signs, cognitive dysfunction and thrombocytopenia could also be due to the underlying conditions. 
Miglustat je ispitivan u indikacijama kod kojih su neki događaji zabilježeni kao nuspojave, poput neuroloških i neurofizioloških simptoma/znakova, kognitivne disfunkcije i trombocitopenije mogli biti i posljedica bolesti od koje se bolesnik liječi.
Pharmacokinetic data were obtained in paediatric patients with type 3 Gaucher disease aged 3 to 15 years, and patients with Niemann-Pick type C disease aged 5-16 years. 
Farmakokinetički podatci dobiveni su od pedijatrijskih bolesnika s Gaucherovom bolesti tipa 3 u dobi od 3 do 15 godina i bolesnika s Niemann-Pickovom bolesti tipa C u dobi od 5 do 16 godina.