natgillin/translations-raw huggingface dataset:
Withdrawal of the marketing authorisation application for Prohippur (sodium benzoate) EMA/258015/2018 
(natrijev benzoat) EMA/258015/2018
What did the company present to support its application? 
Što je tvrtka dostavila u prilog svojem zahtjevu?
The active substance in Prohippur, sodium benzoate, helps reduce harmful levels of glycine by attaching to glycine and forming a substance that is more easily removed by the kidneys. 
Djelatna tvar u lijeku Prohippur, natrijev benzoat, pomaže smanjiti štetne koncentracije glicina tako što se pričvrsti na glicin i stvori tvar koju se lakše ukloni iz bubrega.
• urea cycle disorders, a group of conditions where abnormally high levels of nitrogen occur in the body in the form of ammonia. 
• poremećaja ciklusa ureje, skupine stanja pri kojima se u tijelu pojave nenormalno visoke koncentracije dušika u obliku amonijaka.
• non-ketotic hyperglycinaemia, where abnormally high levels of the amino acid glycine occur in the blood. 
• neketotične hiperglicinemije, pri kojoj se u krvi pojave nenormalno visoke koncentracije aminokiseline glicin
What was Prohippur expected to be used for? 
Za što se Prohippur trebao koristiti?
30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom27 April 2018 EMA/258015/2018 EMEA/H/C/004150 
30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom27. travnja 2018.
The company presented data from published studies on the use of sodium benzoate in patients with non-ketotic hyperglycinaemia and urea cycle disorders. 
Tvrtka je dostavila podatke objavljenih ispitivanja o primjeni natrijeva benzoata u bolesnika s neketotičnom hiperglicinemijom i poremećajima ciklusa ureje.
Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Prohippur did not outweigh its risks. 
U skladu s navedenim, CHMP je u vrijeme povlačenja smatrao kako koristi od lijeka Prohippur ne nadmašuju s njim povezane rizike.
How does Prohippur work? 
Kako djeluje Prohippur?
Further information on the orphan designation can be found here. 
Dodatne informacije o uvrštenju lijekova za rijetke bolesti možete pronaći ovdje
5 years. 
5 godina.
Take the next capsule at the usual time. 
Uzmite sljedeću kapsulu u uobičajeno vrijeme.
The average half-life is 6-7 hours. 
Prosječni poluvijek je 6-7 sati.
This leaflet was last revised in: 
Ova uputa je zadnji puta revidirana u:
Ask your pharmacist how to throw away medicines you no longer take. 
Pitajte svog ljekarnika kako baciti lijekove koje više ne uzimate.
The other ingredients are: 
Drugi sastojci su:
Don't stop taking Zavesca without talking to your doctor. 
Nemojte prestati uzimati Zavescu bez razgovora s liječnikom.
Zavesca can be taken with or without food. 
Zavesca se može uzeti s hranom ili bez nje.
Read all of this leaflet carefully before you start taking this medicine because it contains important information for you. 
Pažljivo pročitajte cijelu uputu prije nego počnete primjenjivati ovaj lijek jer sadrži Vama važne podatke.
What Zavesca is and what it is used for 
Što je Zavesca i za što se koristi
Adult 
Odrasli
Always take this medicine exactly as your doctor has told you. 
Uvijek primjenjujte ovaj lijek točno onako kako Vam je savjetovao liječnik.
They may lower the amount of Zavesca in your body. 
To može sniziti količinu Zavesce u Vašem tijelu.
The greatest prevalence was observed between 6 and 12 months. 
Najveća prevalencija primijećena je između 6 i 12
If you take more Zavesca than you should 
Ako uzmete više Zavesce nego što ste trebali
Date of first authorisation: 20 November 2002 Date of latest renewal: 20 November 2012 
Datum posljednje obnove odobrenja: 20. studenoga 2012.
Capsule, hard 84 capsules 
Kapsula, tvrda 84 kapsule
- if you are allergic to miglustat or any of the other ingredients of this medicine (listed in section 6) 
- ako ste alergični na miglustat ili bilo koji drugi sastojak ovog lijeka (popis u dijelu 6)
Management In case of overdose general medical care is recommended. 
U slučaju predoziranja preporučuje se opća medicinska skrb.
Recommended dose 200 mg three times a day 200 mg twice a day 100 mg three times a day 100 mg twice a day 100 mg once a day 
200 mg triput dnevno 200 mg dvaput dnevno 100 mg triput dnevno 100 mg dvaput dnevno 100 mg jednom dnevno
At present, there is insufficient clinical experience in these patients to provide dosing recommendations. 
Trenutno nema dovoljno kliničkog iskustva s takvim bolesnicima za davanje preporuka za doziranje.
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.
You should not take Zavesca if you are pregnant or thinking of becoming pregnant. 
Zavescu ne smijete uzeti ako ste trudni ili ako namjeravate zatrudniti.
Patients with chronic diarrhoea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice. 
Bolesnike s kroničnim proljevom ili drugim perzistirajućim gastrointestinalnim pojavama koji ne reagiraju na te intervencije treba pregledati u skladu s kliničkom praksom.
If you do lose some weight when you start treatment with Zavesca don't worry. 
Ako izgubite na težini kad počnete liječenje Zavescom, ne brinite.
Weight loss has been reported in 55% of patients. 
U 55 % bolesnika zabilježen je gubitak tjelesne težine.
The tests will help the doctor decide whether these effects are due to your disease or other existing conditions, or due to side effects of Zavesca (see section 4 for further details). 
Ove pretrage će pomoći liječniku ustanoviti jesu li ti učinci posljedica bolesti ili drugih postojećih bolesti ili su posljedica nuspojava Zavesce (za daljnje pojedinosti vidjeti dio 4).
Do not give this medicine to children and adolescents (below 18 years old) with type 1 Gaucher disease because it is not known if it works in this disease. 
Ovaj lijek nemojte davati djeci ni adolescentima (mlađim od 18 godina) s Gaucherovom bolesti tipa 1 jer nije poznato djeluje li kod te bolesti.
Tremor usually began within the first month, and in many cases resolved during treatment after between 1 and 3 months. 
Tremor obično počinje u prvom mjesecu, a u mnogo slučajeva prestaje tijekom liječenja između 1. i 3. mjeseca.
For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder. 
Za sve informacije o ovom lijeku obratite se lokalnom predstavniku nositelja odobrenja za stavljanje lijeka u promet
These findings were considered to be secondary to debilitation. 
Ovi se nalazi smatraju sekundarnima za onesposobljenje.
Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or higher dose. 
Leukopenija i neutropenija su također primijećene u sličnoj skupini bolesnika koji su primali dozu od 800 mg/dan ili višu.
Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Zavesca. 
Obavijestite svog liječnika ako uzimate lijekove koji sadrže imiglucerazu, koji se ponekad uzimaju istovremeno sa Zavescom.
Contents of the pack and other information What Zavesca contains 
Sadržaj pakiranja i druge informacije Što Zavesca sadrži
Titanium dioxide (E171). 
Titanijev dioksid (E171).
There are no adequate data from the use of miglustat in pregnant women. 
Nema dovoljno podataka o primjeni miglustata u trudnica.
What you need to know before you take Zavesca Do not take Zavesca 
Što morate znati prije nego počnete uzimati Zavescu
Other medicines and Zavesca 
Drugi lijekovi i Zavesca
Pharmacotherapeutic group: Other alimentary tract and metabolism products, ATC Code: A16AX06 
Farmakoterapijska skupina: Drugi lijekovi za bolesti probavnog trakta i metabolizma, ATK oznaka: A16AX06
Do not take this medicine after the expiry date which is stated on the carton after ‘EXP'. 
Ovaj lijek se ne smije upotrijebiti nakon isteka roka valjanosti navedenog na kutiji iza „EXP“.
29 patients continued in the extension period. 
29 bolesnika nastavilo je u produljenom razdoblju.
If you stop taking Zavesca 
Ako prestanete uzimati Zavescu
He or she may reduce the dose of Zavesca or recommend other medicines to help control side effects. 
On ili ona može smanjiti dozu Zavesce ili preporučiti drugi lijek koji bi pomogao kontrolirati te nuspojave.
Miglustat did not show any potential for mutagenic or clastogenic effects in the standard battery of genotoxicity tests. 
Miglustat nije pokazao nikakav potencijal za mutagene ili klastogene učinke u standardnom nizu testova na genotoksičnost.
Most patients get one or more of these side effects, usually at the start of treatment or at intervals during treatment. 
Kod većine bolesnika dođe do jedne ili više od tih nuspojava, obično na početku liječenja ili u razmacima tijekom liječenja.
Regular monitoring of vitamin B12 level is recommended because of the high prevalence of vitamin B12 deficiency in patients with type 1 Gaucher disease. 
Preporučuje se redovito praćenje razine vitamina B12 zbog visoke prevalencije pomanjkanja vitamina B12 u bolesnika s Gaucherovom bolesti tipa 1.
Concomitant administration of food decreases the rate of absorption (Cmax was decreased by 36% and tmax delayed 2 hours), but has no statistically significant effect on the extent of absorption of miglustat (AUC decreased by 14%). 
Istovremeno uzimanje hrane smanjuje brzinu apsorpcije (Cmax je smanjen za 36% a tmax je 2 sata dulje), ali nema statistički značajnog utjecaja na apsorpciju miglustata (AUC snižena za 14%).
The requirements for submission of periodic safety update reports for this medicinal product are set out in the list of Union reference dates (EURD list) provided for under Article 107c(7) of Directive 2001/83/EC and any subsequent updates published on the European medicines web-portal. 
• Periodička izvješća o neškodljivosti Zahtjevi za podnošenje periodičkih izvješća o neškodljivosti za ovaj lijek definirani su u referentnom popisu datuma EU (EURD popis) predviđenom člankom 107.c stavkom 7. Direktive 2001/83/EZ i svim sljedećim ažuriranim verzijama objavljenima na europskom internetskom portalu za lijekove.
In the first 6 months in patients who were switched to Zavesca, liver and spleen organ volumes and haemoglobin levels were unchanged. 
U prvih 6 mjeseci kod bolesnika koji su prešli na Zavescu, volumeni organa jetre i slezene i razine hemoglobina bili su nepromijenjeni.
This study also indicated that Zavesca has no or limited effect on the pharmacokinetics of imiglucerase. 
To ispitivanje ukazuje također na to da Zavesca nema nikakav ili ima ograničen učinak na farmakokinetiku imigluceraze.
What Zavesca looks like and contents of the pack 
Kako Zavesca izgleda i sadržaj pakiranja
If you forget to take Zavesca 
Ako zaboravite uzeti Zavescu
Your doctor will perform some tests before and during treatment with Zavesca to assess this (see section 2). 
Liječnik će provesti neke pretrage prije i za vrijeme liječenja Zavescom kako bi to procijenio (vidjeti dio 2).
The MAH shall perform the required pharmacovigilance activities and interventions detailed in the agreed RMP presented in Module 1.8.2 of the Marketing Authorisation and any agreed subsequent updates of the RMP. 
Nositelj odobrenja obavljat će zadane farmakovigilancijske aktivnosti i intervencije, detaljno objašnjene u dogovorenom Planu upravljanja rizikom (RMP), koji se nalazi u Modulu 1.8.2 Odobrenja za stavljanje lijeka u promet, te svim sljedećim dogovorenim ažuriranim verzijama RMP-a..
OTHER SPECIAL WARNING(S), IF NECESSARY EXPIRY DATE 
DRUGO(A) POSEBNO(A) UPOZORENJE(A), AKO JE POTREBNO ROK VALJANOSTI
Among the 41 patients enrolled in the trial 14 patients were treated with Zavesca for more than 3 years. 
Od 41 bolesnika uključenih u ispitivanje 14 bolesnika je liječeno Zavescom duže od 3 godine.
Weight loss, decreased appetite 
Gubitak težine, oslabljen tek
Sometimes your doctor will need to reduce the dose or stop Zavesca treatment to stop the tremor. 
Ponekad će Vam liječnik reći da smanjite dozu ili potpuno prekinete liječenje Zavescom kako bi otklonio tremor.
When compared to the measurements at 6 months, disease control was unchanged after 18 and 24 months of Zavesca monotherapy (20 and 6 patients, respectively). 
U usporedbi s mjerenjima provedenim nakon 6 mjeseci, nadzor bolesti bio je nepromijenjen nakon 18 i 24 mjeseca monoterapije Zavescom (20 odnosno 6 bolesnika).
After 3 years of continuous Zavesca treatment, mean reductions in liver and spleen organ volume were 17.5% and 29.6%, respectively. 
Nakon 3 godine neprekidnog liječenja Zavescom, prosječno smanjenje volumena organa jetre i slezene bilo je 17,5% odnosno 29,6%.
Following administration of a single dose of 100 mg 14C-miglustat to healthy volunteers, 83% of the radioactivity was recovered in urine and 12% in faeces. 
Nakon davanja jedne doze od 100 mg 14C-miglustata zdravim dobrovoljcima, 83% radioaktivno označenog lijeka izlučeno je s urinom a 12% fecesu.
Talk to your doctor or pharmacist before taking Zavesca - if you suffer from kidney disease 
Obratite se svom liječniku ili ljekarniku prije nego uzmete Zavescu ako patite od bolesti bubrega ako patite od bolesti jetre
Zavesca has been used in clinical trials at doses ten times higher than the recommended dose: this caused decreases in white blood cells and other side effects similar to those described in section 4. 
Zavesca je korištena u kliničkim ispitivanjima u deset puta većim dozama od preporučenih: to je uzrokovalo smanjenje broja bijelih krvnih zrnaca i druge nuspojave slične onima opisanim u dijelu 4.
The efficacy of Zavesca in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established. 
Djelotvornost Zavesce kod djece i adolescenata u dobi 0 do 17 godina s Gaucherovom bolesti tipa 1 nije ustanovljena.
Capsule shell Gelatin, 
Ovojnica kapsule Želatina,
Box of 4 blister strips, each blister strip containing 21 capsules providing a total of 84 capsules. 
Kutija s 4 blister stripa, svaki blister strip sadrži 21 kapsulu što je ukupno 84 kapsule.
The most abundant metabolite in urine was miglustat glucuronide accounting for 5% of the dose. 
Metabolit u najvećoj količini u urinu bio je miglustat glukuronid koji je činio 5% doze.
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 kod svih vrsta bili su gubitak tjelesne težine i proljev, a pri višim dozama, oštećenje gastrointestinalne sluznice (erozije i ulceracije).
Male patients should maintain reliable contraceptive methods while taking Zavesca (see sections 4.4 and 5.3). 
Muški bolesnici trebaju primjenjivati pouzdane metode kontracepcije dok uzimaju Zavescu (vidjeti dijelove 4.4 i 5.3).
There is no experience with the use of Zavesca in patients over the age of 70. 
Nema iskustava s primjenom Zavesce u bolesnika starijih od 70 godina.
Detailed information on this medicine is available on the European Medicines Agency website: http://www.ema.europa.eu. 
Detaljnije informacije o ovom lijeku dostupne su na internetskoj stranici Europske agencije za lijekove: http://www.ema.europa.eu.
The metabolite accounting for this has not been identified, but may accumulate and reach concentrations exceeding those of miglustat at steady state. 
Metabolit uzročnik toga nije određen, ali se može akumulirati i dosegnuti koncentracije koje nadilaze one u miglustatu pri stanju dinamičke ravnoteže.
PARTICULARS TO APPEAR ON THE OUTER PACKAGING OUTER CARTON 
PODACI NA KOJI SE MORAJU NALAZITI NA VANJSKOM PAKIRANJU KUTIJA
Data to support safety and efficacy of Zavesca in Niemann-Pick type C disease come from a prospective open-label clinical trial and a retrospective survey. 
Podatci koji podupiru sigurnost i djelotvornost Zavesce u Niemann-Pickovoj bolesti tipa C potječu iz prospektivnog otvorenog kliničkog ispitivanja i retrospektivne analize.
Eighteen patients then continued to receive Zavesca under an optional extended treatment protocol. 
Osamnaest bolesnika je tada nastavilo primati Zavescu u neobaveznom protokolu proširenog liječenja.
Blood and lymphatic system disorders Common 
Poremećaji krvi i limfnog sustava
Limited data suggest that co-administration of Zavesca 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 podatci ukazuju na to da istovremena primjena Zavesce 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).
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 prikupljenoj 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.
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.
In vitro, glucosylceramide synthase is inhibited by miglustat with an IC50 of 20-37 µM. 
In vitro, miglustat inhibira glukozilceramid sintazu sa IC50 koji iznosi 20-37 µM.
In addition, inhibitory action on a non-lysosomal glycosylceramidase has been demonstrated experimentally in vitro. 
Osim toga, inhibitorsko djelovanje nelizosomalne glikozilceramidaze eksperimentalno se pokazalo in vitro.
The efficacy and safety of Zavesca has not been specifically evaluated in patients with severe Gaucher disease. 
Djelotvornost i sigurnost Zavesce nije posebno procjenjivana kod bolesnika s teškom Gaucherovom bolesti.
Growth should be monitored in paediatric and adolescent patients during treatment with Zavesca; the benefit/risk balance should be re-assessed on an individual basis for continuation of therapy. 
U tijeku liječenja Zavescom treba pratiti rast pedijatrijskih bolesnika i adolescenata; omjer koristi/rizika treba ponovo individualno procjenjivati za nastavak liječenja.
The survey included a case series of 66 patients treated with Zavesca outside of the clinical trial for a mean duration of 1.5 years. 
Analiza je uključivala seriju slučajeva od 66 bolesnika liječenih Zavescom izvan kliničkog ispitivanja u prosječnom trajanju od 1,5 godine.
Use of Zavesca in patients with severe renal impairment (creatinine clearance < 30 ml/min/1.73 m2) is not recommended. 
Primjena Zavesce kod bolesnika s teškim oštećenjem funkcije bubrega (klirens kreatinina < 30 ml/min/1,73 m2) se ne preporučuje.
Limited data in patients with Fabry disease and impaired renal function showed that CL/F decreases with decreasing renal function. 
Ograničeni podatci za bolesnike s Fabrijevom bolesti i poremećajem funkcije bubrega pokazuju da CL/F opada sa slabljenjem funkcije bubrega.
Capsule, hard White capsules with “OGT 918” printed in black on the cap and “100” printed in black on the body. 
Kapsula, tvrda Bijele kapsule s “OGT 918” otisnutim crnom bojom na kapici i “100” otisnutim crnom bojom na tijelu.
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 poremećaji, uglavnom proljev, bilo u početku liječenja ili na mahove tijekom liječenja (vidjeti dio 4.8).
Zavesca 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). 
Zavesca 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).
Male patients should maintain reliable contraceptive methods while taking Zavesca. 
Muški bolesnici trebaju primjenjivati pouzdane metode kontracepcije dok uzimaju Zavescu.
The most common serious adverse reaction reported with Zavesca treatment in clinical studies was peripheral neuropathy (see section 4.4). 
Najčešća ozbiljna nuspojava zabilježena liječenjem Zavescom u kliničkim ispitivanjima bila je periferna neuropatija (vidjeti dio 4.4).
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.
The systemic exposure at the lowest dose was below or comparable to that observed in humans (based on AUC0-∞) at the recommended human dose. 
Sistemska izloženost pri najnižoj dozi bila je niža ili se mogla usporediti s onom zapaženom kod ljudi (na osnovu
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 Zavesca between meals, and/or to anti-diarrhoeal medicinal products such as loperamide. 
U kliničkoj praksi, primijećene su gastrointestinalne pojave potaknute miglustatom, kao odgovor na individualiziranu prilagodbu prehrane (na primjer smanjeno uzimanje saharoze, laktoze i drugih ugljikohidrata), uzimanje Zavesce između obroka, i/ili na lijekove protiv proljeva poput loperamida.
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 Zavesca 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 Zavesce s hranom ili privremeno smanjenje doze.
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 smjeli upravljati vozilom ili raditi sa strojevima.
ACLAR/ALU blister strips supplied as a box of 4 blister strips, each blister strip containing 21 capsules providing a total of 84 capsules. 
ACLAR/Al blister stripovi pakirani u kutiju s 4 blister stripa, svaki blister strip sadrži 21 kapsulu što je ukupno 84 kapsule.
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 fecesom, odgovaraju 8, 16 i 33/19 puta višim dozama od najviše preporučene doze za ljude (200 mg triput dnevno).
Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1). 
Zavesca se smije koristiti samo u liječenju bolesnika kod kojih enzimska nadomjesna terapija nije prikladna (vidjeti dijelove 4.4 i 5.1).
Black iron oxide (E172) Shellac. 
Željezov oksid, crni (E172) Šelak
Zavesca should not be taken during breast-feeding. 
Zavesca se ne smije uzimati tijekom dojenja.
No patient showed rapid deterioration of type 1 Gaucher disease following the switch to Zavesca monotherapy. 
Niti jedan bolesnik nije pokazao brzo pogoršanje Gaucherove bolesti tipa 1 nakon prelaska na monoterapiju Zavescom.
Overall the data show that treatment with Zavesca can reduce the progression of clinically relevant neurological symptoms in patients with Niemann-Pick type C disease. 
Ukupni podatci pokazuju da liječenje Zavescom može smanjiti progresiju klinički relevantnih neuroloških simptoma u bolesnika s Niemann-Pickove bolesti tipa C.