natgillin/translations-raw huggingface dataset:
This difference was not statistically significant. 
Ta razlika ni bila statistično značilna.
Two patients were positive at only a single time point. 
Dva bolnika sta bila pozitivna le v eni časovni točki.
There are also links to other websites about rare diseases and treatments. 
Obstajajo tudi povezave do drugih spletnih strani o redkih boleznih in zdravljenju.
The educational material should contain: 
Izobraževalno gradivo mora vsebovati:
Children and adults 
Otroci in odrasli
Elderly population (≥ 65 years old) 
Starejša populacija (≥ 65 let)
Aseptic technique should be used. 
Uporabiti je treba aseptično tehniko.
Infants (< 6 months of age) 
Dojenčki (< 6 mesecev starosti)
The age range at study entry was 1-6 months. 
Starostni razpon ob vstopu v študijo je bil od 1 do 6 mesecev.
This is known as an infusion reaction which can sometimes be severe, and may include an allergic reaction. 
To je znano kot reakcija na infuzijo, ki je lahko včasih resna in vključuje alergijsko reakcijo.
Each infusion will take approximately 1 to 2 hours. 
Vsaka infuzija traja približno 1 do 2 uri.
Additional risk minimisation measures 
Dodatni ukrepi za zmanjševanje tveganj
Final study report expected in July 2019 
študiji se pričakuje julija 2019
If you have any further questions, ask your doctor. 
Če imate dodatna vprašanja, se posvetujte z zdravnikom.
These increases have generally occurred within the first 2 to 4 weeks and improved within a further 8 weeks of treatment. 
Ta povečanja so se načeloma pojavila v prvih 2 do 4 tednih in so se v naslednjih 8 tednih zdravljenja izboljšala.
The Guide for healthcare professionals shall contain the following key elements: 
Navodilo za zdravstvene delavce mora vsebovati naslednje ključne elemente:
MARKETING AUTHORISATION NUMBER(S) EU/1/15/1033/001 
ŠTEVILKA(E) DOVOLJENJA (DOVOLJENJ) ZA PROMET EU/1/15/1033/001
KANUMA should be started at as young an age as possible and is intended for long-term use. 
Zdravilo KANUMA je treba začeti dajati kar se da zgodaj v mladosti, in je namenjeno za dolgotrajno uporabo.
DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 28 August 2015 
DATUM PRIDOBITVE/PODALJŠANJA DOVOLJENJA ZA PROMET Z ZDRAVILOM 28. avgust 2015
Unopened vials: 2 years. 
Neodprte viale: 2 leti
With continued treatment beyond 12 months of age, 1 additional patient died at age 15 months. 
Z nadaljevanjem zdravljenja po 12. mesecu starosti je v starosti 15 mesecev umrl še 1 bolnik.
Store in a refrigerator (2°C to 8°C). 
Shranjujte v hladilniku (2 ºC do 8 ºC).
Read all of this leaflet carefully before you are given this medicine because it contains important information for you. 
Pred začetkom dobivanja zdravila natančno preberite navodilo, ker vsebuje za vas pomembne podatke!
Frequency, type and severity of adverse reactions in children are the same as in adults. 
Pogostnost, vrsta in resnost neželenih učinkov pri otrocih so enaki kot pri odraslih.
What KANUMA is and what it is used for 
Kaj je zdravilo KANUMA in za kaj ga uporabljamo
Pre-treatment with antipyretics and/or antihistamines may prevent subsequent reactions in those cases where symptomatic treatment was required. 
Premedikacija z antipiretiki in/ali antihistaminiki lahko prepreči nadaljnje reakcije v primerih, kjer je bilo potrebno simptomatsko zdravljenje.
In the absence of more clinical data, this dose is not recommended. 
Ker več kliničnih podatkov ni na voljo, ta odmerek ni priporočljiv.
Based on the limited data currently available the development of ADA seems to occur more frequently in infants.' 
Na podlagi omejenih podatkov se zdi, da ADA razvije pogosteje pri dojenčkih.
From a microbiological point of view, the diluted solution should be used immediately. 
Z mikrobiološkega vidika je treba razredčeno raztopino porabiti takoj.
Posology It is important to initiate treatment as early as possible after diagnosis of LAL deficiency. 
Odmerjanje Pomembno je, da se zdravljenje uvede čim prej po postavitvi diagnoze pomanjkanja LAL.
Studies to evaluate the mutagenic and carcinogenic potential of sebelipase alfa have not been performed. 
Študij za oceno mutagenega in kancerogenega potenciala sebelipaze alfa niso opravili.
The efficacy objectives are assessment of hepatic function overtime up to 3 years and survival at 12 months. 
Cilji za učinkovitost so ocena delovanja jeter skozi čas do 3 let in preživetje pri 12 mesecih starosti.
Sebelipase alfa has not been investigated in patients 2 to 4 years of age or patients aged 65 years or older. 
Sebelipaze alfa niso preučili pri bolnikih, starih od 2 do 4 let ali pri bolnikih, starih 65 let ali več.
If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine. 
Če ste noseči ali dojite, menite, da bi lahko bili noseči ali načrtujete zanositev, se posvetujte z zdravnikom, preden prejmete to zdravilo.
The age range at randomisation was 4-58 years old (71% were < 18 years old). 
Starostni razpon ob randomizaciji je bil 4 do 58 let (71% jih je bilo starih < 18 let).
Study LAL-CL02 - Children and Adults 1 mg/kg once every other week 
Študija LAL-CL02 - otroci in odrasli 1 mg/kg enkrat vsak drugi teden
* The infusion volume should be based on the prescribed dose and should be prepared to a final sebelipase alfa concentration of 0.1-1.5 mg/ml. 
* Količina za infundiranje mora temeljiti na predpisanem odmerku; pripraviti je treba končno koncentracijo sebelipaze alfa 0,1 do 1,5 mg/ml.
Prior to launch of Kanuma in each Member State the Marketing Authorisation Holder (MAH) must agree about the content and format of the educational material including communication media, distribution modalities, and any other aspects of the programme, with the National Competent Authority. 
Pred začetkom trženja zdravila Kanuma v vsaki od držav članic se mora imetnik dovoljenja za promet z zdravilom dogovoriti s pristojnim nacionalnim organom o vsebini in obliki izobraževalnega gradiva, vključno s komunikacijskimi mediji, načini distribucije, in vsemi drugimi vidiki programa.
Total infusion volume (ml) 
Skupna količina za infundiranje (ml)
4-11 years old N=24 
4 do 11 let starosti n=24
In cases of severe infusion reactions and in cases of lack or loss of effect, patients should be tested for the presence of antibodies. 
V primerih hude reakcije ob infundiranju in v primeru pomanjkanja ali izgube učinka, je treba bolnike testirati na prisotnostprotiteles.
For study entry, patients were required to have ALT levels of ≥1.5 X upper limit of normal (ULN). 
Za vstop v študijo so bolniki morali imeti ravni ALT ≥ 1,5 X zgornja meja normalne vrednosti (ULN-upper limit of normal).
Proportion of patients who achieved normalisation defined as 34-59 U/l, depending on age and gender. 
Delež bolnikov, ki so dosegli normalizacijo, opredeljeno kot 34 do 59 e./l, odvisno od starosti in spola.
• Guide for healthcare professionals 
• navodilo za zdravstvene delavce.
The infusion period may be extended in the event of dose escalation. 
Obdobje infundiranja se lahko v primeru povečanja odmerka podaljša.
The majority of reactions occurred during or within 4 hours of the completion of the infusion. 
Večina reakcij se je pojavila med infundiranjem ali v 4 urah po njem.
Patients also had rapidly progressive liver disease and severe hepatosplenomegaly. 
Bolniki so imeli tudi hitro napredujočo bolezen jeter in resno hepatosplenomegalijo.
The dose you or your child receives is based on your or your child's body weight. 
Odmerek, ki ga bosta vi oziroma vaš otrok prejela, je odvisen od vaše oziroma otrokove telesne mase.
Patients with known egg allergies were excluded from clinical studies (see section 4.3). 
Bolniki z znano alergijo na jajca so bili iz kliničnih študij izključeni (glejte poglavje 4.3).
The vials should not be left outside the refrigerator longer than 24 hours prior to dilution for infusion. 
Vial ni dovoljeno pustiti zunaj hladilnika dlje kot 24 ur pred razredčitvijo za infundiranje.
The association between the development of ADA to sebelipase alfa and reductions in treatment effect or the occurrence of adverse reactions has not been determined. 
Povezava med razvojem ADA na sebelipazo alfa in zmanjšanjem učinka zdravljenja ali pojavom neželenih učinkov ni bila ugotovljena.
B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE Medicinal product subject to restricted medical prescription (see Annex I: Summary of Product Characteristics, section 4.2). 
B. POGOJI ALI OMEJITVE GLEDE OSKRBE IN UPORABE Predpisovanje in izdaja zdravila je le na posebni recept (glejte Prilogo I: Povzetek glavnih značilnosti zdravila, poglavje 4.2).
The risks and benefits of re-administering sebelipase alfa following a severe reaction should be considered. 
Upoštevati je treba koristi in tveganja ponovnega zdravljenja s sebelipazo alfa po hudi reakciji.
• Information to healthcare professionals that it is the responsibility of the MAH to provide the test for the monitoring of ADA positive patients including the modalities for requesting the test. 
• Informacije za zdravstvene delavce o tem, da je odgovornost imetnika dovoljenja za promet z zdravilom zagotoviti preskus za spremljanje ADA-pozitivnih bolnikov, vključno z načini, kako tak preskus zahtevati.
Children and adults with LAL deficiency 
Otroci in odrasli s pomanjkanjem LAL
12-17 years old N=23 
12 do 17 let starosti n=23
This medicine may contain egg proteins. 
Zdravilo lahko vsebuje jajčne beljakovine.
Of the 32 patients who had a liver biopsy at study entry, 100% had fibrosis and 31% had cirrhosis. 
Od 32 bolnikov, pri katerih so ob vstopu v študijo opravili biopsijo jeter, jih je imelo 100% fibrozo in 31% cirozo.
What you need to know before KANUMA is given 
Kaj morate vedeti, preden boste dobili zdravilo KANUMA
This means that it replaces the missing or defective LAL enzyme in patients with LAL deficiency. 
To pomeni, da nadomesti manjkajoči ali okvarjeni encim LAL pri bolnikih s pomanjkanjem LAL.
The age range of patients with biopsy evidence of cirrhosis was 4-21 years old. 
Starostni razpon bolnikov s cirozo, dokazano z biopsijo, je bil od 4 do 21 let.
Open-label period 
Odprto obdobje
Table 3: Primary and secondary efficacy endpoints in LAL-CL02 Endpoint 
Preglednica 3: Primarni in sekundarni opazovani dogodki učinkovitosti v študiji LAL-CL02
There are no data from studies in breast-feeding women. 
Ni podatkov iz študij z ženskami, ki dojijo.
The safety objectives should focus on hypersensitivity reactions, particularly anti-drug antibodies development impacting response to drug. 
Cilji za varnostni bi se morali osredotočiti na preobčutljivostne reakcije, zlasti razvoj protiteles proti zdravilu, ki vplivajo na odziv na zdravilo.
You should not be given KANUMA if you are pregnant unless clearly necessary. 
Če ste noseči, zdravila KANUMA ne smete dobiti, razen če je nujno potrebno.
Following the first sebelipase alfa infusion, including the first infusion after a dose escalation, patients should be observed for 1 hour in order to monitor for any signs or symptoms of anaphylaxis or a severe hypersensitivity reaction. 
Po prvem infundiranju sebelipaze alfa, vključno s prvo infuzijo po povečanjku odmerka, je treba bolnike opazovati 1 uro in spremljati morebitne znake ali simptome anafilaksije ali hudih preobčutljivostnih reakcij.
Twenty-four patients were aged 411 years, 23 were aged 12-17 years, and 18 were aged ≥ 18 years (Table 4). 
Štiriindvajset bolnikov je bilo starih od 4 do 11 let, 23 jih je bilo starih od 12 do 17 let in 18 jih je bilo starih ≥ 18 let
The European Medicines Agency has deferred the obligation to submit the results of studies with KANUMA in one or more subsets of the paediatric population in LAL deficiency (see section 4.2 for information on paediatric use). 
Evropska agencija za zdravila je začasno odložila zahtevo za predložitev rezultatov študij z zdravilom KANUMA za eno ali več podskupin pediatrične populacije s pomanjkanjem LAL (za podatke o uporabi pri pediatrični populaciji glejte poglavje 4.2).
The absolute reduction in mean ALT level was -57.9 U/l (-53%) in the sebelipase alfa-treated group and -6.7 U/l (-6%) in the placebo group. 
Absolutno zmanjšanje povprečne ravni ALT je bilo -57,9 e./l (-53%) v skupini, zdravljeni s sebelipazo alfa, in -6,7 e./l (-6%) v skupini s placebom.
For management of adverse reactions, see sections 4.4 and 4.8. 
Za obvladovanje neželenih učinkov glejte poglavji 4.4 in 4.8.
KANUMA (n = 36) 
Zdravilo KANUMA (n=36)
It is not known whether sebelipase alfa passes into breast milk; therefore it is recommended that you stop breast-feeding or using KANUMA treatment while breast-feeding. 
Ni znano, ali sebelipaza alfa prehaja v materino mleko, zato priporočamo, da med dojenjem prenehate dojiti ali uporabljati zdravilo KANUMA.
These medicines may include antihistamines, fever-reducing medicines and/or corticosteroids (a type of antiinflammatory medicines). 
Ta zdravila lahko vključujejo antihistaminike, zdravila za zbijanje zvišane telesne temperature in/ali kortikosteroide (vrsta protivnetnih zdravil).
c. The vials should not be shaken. 
c. Vial ni dovoljeno stresati.
Pharmacotherapeutic group: Other alimentary tract and metabolism products, Enzymes; ATC code: not yet assigned 
Farmakoterapevtska skupina: druga zdravila za bolezni prebavil in presnove, encimi; oznaka ATC: še ni dodeljena
KANUMA should be administered by a trained healthcare professional who can manage medical emergencies. 
Zdravilo KANUMA mora dajati usposobljen zdravstveni delavec, ki obvlada obravnavo nujnih zdravstvenih primerov.
Of patients with paired liver biopsies, 63% (10/16) of KANUMA-treated patients had improvement in hepatic steatosis (at least ≥ 5% reduction) as measured by morphometry compared to 40% (4/10) of placebo patients. 
Pri bolnikih s parnimi jetrnimi biopsijami se je pri 63% (10/16) bolnikov, zdravljenih z zdravilom KANUMA, jetrna steatoza izboljšala (vsaj ≥ 5-% zmanjšanje), izmerjeno z morfometrijo, v primerjavi s 40% (4/10) bolnikov s placebom.
KANUMA should be administered through a 0.2 μm filter (see section 6.6). 
Zdravilo KANUMA je treba dati skozi filter velikosti 0,2 μm (glejte poglavje 6.6).
Adverse reactions are listed by System Organ Class and frequency. 
Neželeni učinki so navedeni po organskih sistemih in pogostnosti.
The most serious adverse reactions experienced by 3% of patients in clinical studies were signs and symptoms consistent with anaphylaxis. 
Najresnejši neželeni učinki, ki so jih doživeli 3% bolnikov v kliničnih študijah, so bili znaki in simptomi, skladni z anafilakso.
From baseline to week 48, mean weight-for-age percentile improved from 12.74% to 29.83% and mean serum albumin levels increased from 26.7 g/l to 38.7 g/l. 
Od izhodišča do 48. tedna se je povprečni percentil telesne mase glede na starost izboljšal z 12,74% na 29,83%, povprečne ravni serumskega albumina pa so se povečale s 26,7 g/l na 38,7 g/l.
The total volume of the infusion should be administered over approximately 2 hours. 
Skupno količino infuzije je treba dati v približno 2-urnem obdobju.
PHARMACEUTICAL FORM AND CONTENTS Concentrate for solution for infusion 1 vial of 10 ml 
FARMACEVTSKA OBLIKA IN VSEBINA koncentrat za raztopino za infundiranje 1 viala z 10 ml
For patients who have experienced allergic reactions during infusion, caution should be exercised upon re-administration. 
Pri bolnikih, ki so med infundiranjem imeli alergijske reakcije, je potrebna previdnost pri ponovnem dajanju.
The safety and efficacy of KANUMA in overweight patients have not been thoroughly evaluated and therefore no alternative dose regimens can be recommended for these patients at this time. 
Varnost in učinkovitost zdravila Kanuma pri bolnikih s prekomerno telesno maso ni bila celovito ocenjena, zato za to skupino bolnikov trenutno ni mogoče priporočiti alternativnih režimov odmerjanja.
In addition to liver disease, patients with LAL deficiency experience increased risk for cardiovascular disease and accelerated atherosclerosis. 
Poleg bolezni jeter se pri bolnikih s pomanjkanjem LAL pojavi večje tveganje za srčno-žilna obolenja in pospešeno aterosklerozo.
Side effects were seen while patients were being given the medicine or shortly after (infusion reactions). 
Neželeni učinki so se pojavili pri bolnikih med prejemanjem zdravila ali tik po tem (reakcija na infuzijo).
This medicinal product may contain traces of egg proteins. 
To zdravilo lahko vsebuje sledove jajčnih beljakovin.
The difference in exposures to sebelipase alfa between the once weekly 0.35 mg/kg and 3 mg/kg groups was more than dose proportional, with a 8.6-fold increase in dose resulting in a 9.6-fold increase in exposure for AUC and a 10.0-fold increase for Cmax. 
Razlika v izpostavljenostih sebelipazi alfa v skupini 0,35 mg/kg enkrat na teden in 3 mg/kg je bila več kot sorazmerna z odmerkom, z 8,6kratnim povečanjem odmerka, kar je povzročilo 9,6-kratno povečanje izpostavljenosti za AUC in 10,0-kratno povečanje za Cmax.
• Warning and precautions on the the risk of hypersensitivity including anaphylaxis or ADA development, with particular reference to symptoms, time to onset and severity. 
• opozorilo in previdnostne ukrepe glede tveganja preobčutljivosti, vključno z anafilaksijo, ali za razvoj ADA, s posebnim poudarkom na simptomih, času do pojava in resnosti.
One infant was treated with 5 mg/kg once weekly in LAL-CL03; no new adverse reactions were reported at this dose. 
En dojenček je bil zdravljen s 5 mg/kg enkrat na teden pri LAL-CL3; o novih neželenih učinkih pri tem odmerku niso poročali.
Alexion Europe SAS 103-105 rue Anatole France 92300 Levallois-Perret France 
Alexion Europe SAS 103-105 rue Anatole France 92300 Levallois-Perret Francija
LAL-CL02 was a multicentre, double-blind, placebo-controlled study in 66 children and adults with LAL deficiency. 
LAL-CL02 je bila multicentrična, dvojno slepa, s placebom nadzorovana študija pri 66 otrocih in odraslih s pomanjkanjem LAL.
Pharmacokinetic Parameter 
Farmakokinetični parametri
LAL deficiency presenting in infants is a medical emergency with rapid disease progression over a period of weeks that is typically fatal within the first 6 months of life. 
Pomanjkanje LAL pri dojenčkih je nujno zdravstveno stanje s hitrim napredovanjem bolezni v obdobju nekaj tednov, ki se običajno konča s smrtjo v prvih 6 mesecih življenja.
Tell your doctor if you or your child is on a controlled sodium diet. 
Povejte svojemu zdravniku, če sta vi oziroma vaš otrokna dieti z nadzorovanim vnosom natrija.
Pack sizes: 1 vial containing 10 ml of concentrate. 
Velikosti pakiranja: 1 viala, ki vsebuje 10 ml koncentrata.
Almac Pharma Services Seagoe Industrial Estate Craigavon BT63 5UA United Kingdom 
Almac Pharma Services Seagoe Industrial Estate Craigavon BT63 5UA Velika Britanija
• Information on the ongoing LAL Deficiency Registry, including the importance of enrolling patients, also those not treated with Kanuma, and the modalities for participation. 
Informacije o aktualnem Registru o pomanjkanju LAL, vključno s pomenom vpisovanja bolnikov, tudi tistih, ki niso zdravljeni z zdravilom Kanuma, in o načinih udeležbe.
The majority of patients (58%) had LDLcholesterol > 190 mg/dl at study entry, and 24% of patients with LDL-cholesterol > 190 mg/dl were on lipid lowering medicinal products. 
Večina bolnikov (58%) je imela ob vstopu v študijo holesterol LDL > 190 mg/dl in 24% bolnikov, ki je imelo holesterol LDL > 190 ml/dl je jemalo zdravila za zmanjševanje maščob.
• Information on how to manage patients experiencing severe hypersensitivity reactions including anaphylaxis. 
• Informacije o tem, kako ravnati z bolniki, ki doživijo hude preobčutljivostne reakcije, vključno z anafilaksijo.
Preparation of the sebelipase alfa infusion KANUMA should be prepared and used according to the following steps. 
Priprava infuzije sebelipaze alfa Zdravilo KANUMA je treba pripraviti in uporabiti v skladu z naslednjimi koraki.
Table 4: Mean Population Pharmacokinetic Parameters 
Preglednica 4: Povprečni parametri populacijske farmakokinetike
Medical or healthcare professionals are encouraged to participate and enrol all patients diagnosed with LAL deficiency in the LAL deficiency registry. 
Medicinske oziroma zdravstvene delavce spodbujamo k sodelovanju in vpisu vseh bolnikov z diagnozo pomanjkanja LAL v register o pomanjkanju LAL.
The safety and efficacy of KANUMA in patients older than 65 years have not been evaluated and no alternative dose regimens can be recommended for these patients. 
Varnost in učinkovitost zdravila KANUMA pri bolnikih, starejših od 65 let, nista bili ovrednoteni, zato priporočil o drugačnem režimu odmerjanja pri teh bolnikih ni mogoče dati.
A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from sebelipase alfa therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman. 
Odločiti se je treba med prenehanjem dojenja in prenehanjem/prekinitvijo zdravljenja s sebelipazo alfa, pri čemer je treba pretehtati prednosti dojenja za otroka in prednosti zdravljenja za mater.
Following the occurrence of a hypersensitivity reaction, appropriate pre-treatment should be considered according to the standard of care (see section 4.4). 
Po pojavu preobčutljivostne reakcije je treba razmisliti o ustrezni premedikaciji v skladu s standardno nego (glejte poglavje 4.4).
Sebelipase alfa is a protein and is expected to be metabolically degraded through peptide hydrolysis. 
Sebelipaza alfa je beljakovina in pričakuje se, da se bo s presnovo razgradila s pomočjo peptidne hidrolize.
The management of hypersensitivity reactions may include temporarily interrupting the infusion, lowering the infusion rate, and/or treatment with antihistamines, antipyretics, and/or corticosteroids. 
Obvladovanje preobčutljivostne reakcije lahko vključuje začasno prekinitev infundiranja, zmanjšanje infuzijske hitrosti in/ali zdravljenje z antihistaminiki, antipiretiki in/ali kortikosteroidi.
It is not known whether sebelipase alfa is excreted in human milk. 
Ni znano, ali se sebelipaza alfa izloča v materino mleko.
Most patients who developed ADA did so within the first 2 months of exposure. 
Pri večini bolnikov se je ADA razvila v prvih 2 mesecih izpostavljenosti.
All patients were receiving 1 mg/kg once every other week. 
Vsi bolniki so dobivali 1 mg/kg enkrat vsak drugi teden.
LAL deficiency is a rare disease associated with significant morbidity and mortality, which affects individuals from infancy through adulthood. 
Pomanjkanje LAL je redka bolezen, povezana z znatno obolevnostjo in smrtnostjo, ki prizadene posameznike od dojenčkov do odraslih.
Chronic toxicity studies in juvenile cynomolgous monkeys showed no toxicity at doses up to 3 times the recommended dose in infants and 10 times the recommended dose in adults/children. 
Študije kronične toksičnosti pri juvenilnih opicah cynomolgous niso pokazale toksičnosti pri odmerkih, ki so bili do 3-krat večji od priporočenega odmerka pri dojenčkih in 10-krat večji od priporočenega odmerka pri odraslih/otrocih.
In the historical cohort, 0 of 21 patients survived beyond 8 months of age (0% 12-month survival, 95% CI: 0% to 
V zgodovinski kohorti ni nobeden od 21 bolnikov preživel več kot 8 mesecev (0% 12-mesečno preživetje, 95% IZ: 0% do 16%).
This medicine works by lowering the build-up of fat that causes medical complications, including impaired growth, liver damage and heart complications. 
To zdravilo deluje tako, da zmanjšuje kopičenje maščobe, ki povzroča zdravstvene zaplete, vključno z moteno rastjo, poškodbo jeter in zapleti na srcu.
Frequency a 
Pogostnost a
In clinical studies, doses of sebelipase alfa were explored up to 5 mg/kg once weekly and no specific signs or symptoms were identified following the higher doses. 
V kliničnih preskušanjih so preučili odmerke sebelipaze alfa do 5 mg/kg enkrat na teden, pri višjih odmerkih pa niso opazili specifičnih znakov ali simptomov.