FrancophonIA/ELRC3.0 huggingface dataset:
English: • Details on how to monitor for potential ADA formation following initiation of treatment with Kanuma, particularly in patients on Kanuma who experience clinically important hypersensitivity reactions or potential loss of clinical response.
Croatian: • Pojedinosti o nadziranju mogućeg nastanka ADA-a nakon započinjanja liječenja Kanumom, posebice u bolesnika koji primaju Kanumu u kojih su se razvile klinički značajne reakcije preosjetljivosti ili moguć gubitak kliničkog odgovora.
English: The most serious side effects may include an allergic reaction (seen very commonly [may affect more than 1 in 10 people] in infants younger than 6 months old, or commonly [may affect up to 1 in 10 people] in children and adults) with symptoms including difficulty breathing, swelling of the throat, rapid breathing, fast heartbeat, chest discomfort, mild swelling of eyelids, red eyes, runny nose, flushing, and hives.
Croatian: Najozbiljnije nuspojave mogu uključivati alergijsku reakciju (uočena vrlo često [može se javiti u više od 1 na 10 osoba] u dojenčadi mlađe od 6 mjeseci, ili često [može se javiti u do 1 na 10 osoba] u djece i odraslih) sa simptomima koji uključuju otežano disanje, oticanje grla, ubrzano disanje, ubrzane otkucaje srca, nelagodu u prsištu, blago oticanje vjeđa, crvenilo očiju, curenje nosa, navale crvenila i koprivnjaču.
English: • Warning and precautions on the the risk of hypersensitivity including anaphylaxis or ADA development, with particular reference to symptoms, time to onset and severity.
Croatian: • Upozorenje i mjere opreza o riziku od preosjetljivosti, uključujući anafilaksiju, ili razvoj ADA-a, s posebnim osvrtom na simptome, vrijeme do nastanka i težinu.
English: d. Do not use if the solution is cloudy, or if foreign particulate matter is present.
Croatian: d. Nemojte primijeniti otopinu ako je zamućena ili ako su u njoj prisutne strane čestice.
English: Clear glass vial (Type I) with a siliconised butyl rubber stopper, and an aluminium seal with a plastic flip-off cap, containing 10 ml of concentrate.
Croatian: Prozirna staklena bočica (tip I) sa silikoniziranim čepom od butilne gume i aluminijskom zaštitom s plastičnim flip-off zatvaračem koja sadrži 10 ml koncentrata.
English: Anaphylaxis occurred during the infusion as late as 1 year after treatment initiation.
Croatian: Anafilaksija se javljala tijekom infuzije čak i do godinu dana od započinjanja liječenja.
English: This medicine works by lowering the build-up of fat that causes medical complications, including impaired growth, liver damage and heart complications.
Croatian: Ovaj lijek djeluje tako što smanjuje nakupljanje masnoća koje uzrokuje zdravstvene komplikacije, uključujući usporeni rast, oštećenje jetre i srčane probleme.
English: Hypersensitivity reactions including anaphylaxis
Croatian: Reakcije preosjetljivosti uključujući anafilaksiju
English: The safety objectives should focus on hypersensitivity reactions, particularly anti-drug antibodies development impacting response to drug.
Croatian: Ciljevi sigurnosti trebaju biti usredotočeni na reakcije preosjetljivosti, posebice na razvoj protutijela na lijek i njihov utjecaj na odgovor na lijek.
English: 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.
Croatian: Razlika u izloženosti sebelipazi alfa između skupina koje su primale 0,35 mg/kg i 3 mg/kg jednom tjedno bila je veća nego proporcionalna dozi; povećanje doze za 8,6 puta izazvalo je povećanje izloženosti od 9,6 puta za AUC i povećanje od 10,0 puta za Cmax.
English: Placebo patients had persistently elevated serum transaminase and abnormal serum lipid levels during the double-blind period.
Croatian: Bolesnici koji su primali placebo imali su stalno povišene serumske transaminaze i abnormalne razine serumskih lipida tijekom dvostruko slijepog razdoblja.
English: Dose escalation to 3 mg/kg once weekly was associated with additional improvements in weight gain, lymphadenopathy and serum albumin.
Croatian: Povećanje doze na 3 mg/kg jednom tjedno bilo je povezano s dodatnim poboljšanjima dobitka na težini, limfadenopatije i serumskog albumina.
English: 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.
Croatian: Od bolesnika s uparenim uzorcima biopsije jetre, u 63% (10/16) bolesnika liječenih KANUMOM došlo je poboljšanja jetrene steatoze (najmanje ≥ 5% smanjenja) u usporedbi s 40% (4/10) bolesnika koji su primali placebo, mjereno morfometrijom.
English: Prior to dilution, the solution in the vials should be inspected visually; the solution should be clear to slightly opalescent, colourless to slightly coloured (yellow).
Croatian: Prije razrjeđivanja otopinu u bočicama treba vizualno pregledati; otopina treba biti bistra do blago opalescentna, bezbojna do blago obojena (žuta).
English: All patients were receiving 1 mg/kg once every other week.
Croatian: Svi su bolesnici primali 1 mg/kg jednom svaki drugi tjedan.
English: What KANUMA looks like and contents of the pack KANUMA is supplied as a concentrate for solution for infusion.
Croatian: Kako KANUMA izgleda i sadržaj pakiranja KANUMA je dostupna kao koncentrat za otopinu za infuziju.
English: If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user and would normally not be longer than 24 hours at 2°C to 8°C, or up to 12 hours below 25°C, unless dilution has taken place in controlled and validated aseptic conditions.
Croatian: Ako se ne primijeni odmah, vrijeme i uvjeti čuvanja prije primjene odgovornost su korisnika i normalno to ne bi smjelo biti duže od 24 sata pri temperaturi od 2 °C do 8 °C ili do 12 sati pri temperaturi ispod 25 °C, osim ako je razrjeđivanje provedeno u kontroliranim i validiranim aseptičnim uvjetima.
English: Those patients who developed ADA did so within the first 3 months of exposure.
Croatian: U bolesnika u kojih su se razvila ADA to se dogodilo unutar prva 3 mjeseca izloženosti.
English: One infant was treated with 5 mg/kg once weekly in LAL-CL03; no new adverse reactions were reported at this dose.
Croatian: Jedno dojenče bilo je liječeno dozom od 5 mg/kg jednom tjedno u ispitivanju LAL-CL03; nisu zabilježene nove nuspojave pri ovoj dozi.
English: If severe reactions occur, the sebelipase alfa infusion should be immediately stopped and appropriate medical treatment should be initiated.
Croatian: Ako se razviju teške reakcije, infuzija sebelipaze alfa se mora odmah prekinuti i započeti odgovarajuće liječenje.
English: Medical or healthcare professionals are encouraged to participate and enrol all patients diagnosed with LAL deficiency in the LAL deficiency registry.
Croatian: Medicinski i zdravstveni radnici potiču se na sudjelovanje i uključivanje svih bolesnika s dijagnozom nedostatka LAL-a u registar nedostatka LAL-a.
English: For patients who have experienced allergic reactions during infusion, caution should be exercised upon re-administration.
Croatian: U bolesnika u kojih je došlo do alergijske reakcije tijekom infuzije, pri ponovnoj primjeni potreban je oprez.
English: No dosing adjustment is recommended in patients with renal or hepatic impairment based on current knowledge of the pharmacokinetics and pharmacodynamics of sebelipase alfa.
Croatian: Nema preporuka o prilagodbi doziranja u bolesnika s oštećenjem bubrega ili jetre temeljeno na sadašnjem znanju o farmakokinetici i farmakodinamici sebelipaze alfa.
English: The most serious adverse reactions experienced by 3% of patients in clinical studies were signs and symptoms consistent with anaphylaxis.
Croatian: Najozbiljnije nuspojave koje su se javile u 3% bolesnika tijekom kliničkih ispitivanja bile su znakovi i simptomi u skladu s anafilaksijom.
English: Proportion of patients who achieved normalisation defined as 34-59 U/l, depending on age and gender.
Croatian: b Udio bolesnika koji su postigli normalizaciju definiranu kao 34-59 U/l, ovisno o dobi i spolu.
English: No accumulation was seen at 1 mg/kg (once weekly or once every other week) or 3 mg/kg once weekly.
Croatian: Nije uočena akumulacija pri dozi od 1 mg/kg (jednom tjedno ili jednom svaki drugi tjedan) ili 3 mg/kg jednom tjedno.
English: Life-threatening hypersensitivity (anaphylactic reaction) to the active substance when attempts to rechallenge are unsuccessful, or to egg or any of the excipients listed in section 6.1, (see section 4.4).
Croatian: Životno ugrožavajuća preosjetljivost (anafilaktička reakcija) na djelatnu tvar kad pokušaji ponovnog uvođenja lijeka nisu uspjeli ili preosjetljivost na jaje ili neku od pomoćnih tvari navedenih u dijelu 6.1, (vidjeti dio 4.4).
English: 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.
Croatian: Sigurnost i djelotvornost KANUME u bolesnika s prekomjernom tjelesnom težinom nisu temeljito procijenjene te se stoga za sada ne mogu preporučiti alternativni režimi doziranja za ove bolesnike.
English: The management of hypersensitivity reactions may include temporarily interrupting the infusion, lowering the infusion rate, and/or treatment with antihistamines, antipyretics, and/or corticosteroids.
Croatian: Zbrinjavanje reakcija preosjetljivosti može uključivati privremeno prekidanje infuzije, smanjenje brzine infuzije i/ili liječenje antihistaminicima, antipireticima i/ili kortikosteroidima.
English: If you or your child experiences a severe infusion reaction like this, seek immediate medical attention.
Croatian: Ako Vi ili Vaše dijete doživite tešku infuzijsku reakciju poput ove, odmah potražite liječničku pomoć.
English: Patients have developed anti-drug antibodies (ADA) to sebelipase alfa.
Croatian: Bolesnici su razvili protutijela na lijek (engl. anti-drug antibody - ADA) na sebelipazu alfa.
English: Patients were randomised to receive KANUMA at a dose of 1 mg/kg (n = 36) or placebo (n = 30) once every other week for 20 weeks in the double-blind period.
Croatian: Bolesnici su randomizirani kako bi primali KANUMU u dozi od 1 mg/kg (n=36) ili placebo (n=30) jednom svaki drugi tjedan tijekom 20 tjedana u dvostruko slijepom razdoblju.
English: In LAL-CL03, sebelipase alfa was eliminated from the systemic circulation with a median T1/2 of 0.1 hr (range: 0.1-0.2) at the 3 mg/kg once weekly dose (n = 4).
Croatian: U ispitivanju LAL-CL03 KANUMA je bila eliminirana iz sistemske cirkulacije s medijanom T1/2 od 0,1 h (raspon: 0,1-0,2) pri dozi od 3 mg/kg jednom tjedno (n=4).
English: PHARMACEUTICAL FORM AND CONTENTS Concentrate for solution for infusion 1 vial of 10 ml
Croatian: OBLIK I SADRŽAJ Koncentrat za otopinu za infuziju 1 bočica od 10 ml
English: Driving and using machines KANUMA has no or negligible influence on the ability to drive and use machines.
Croatian: Upravljanje vozilima i strojevima KANUMA ne utječe ili zanemarivo utječe na sposobnost upravljanja vozilima i rada sa strojevima.
English: 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.
Croatian: Nije poznato izlučuje li se sebelipaza alfa u majčino mlijeko te se stoga preporučuje prekinuti dojenje ili liječenje KANUMOM dok dojite.
English: CONTENTS BY WEIGHT, BY VOLUME OR BY UNIT 20 mg/10 ml
Croatian: SADRŽAJ PO TEŽINI, VOLUMENU ILI DOZNOJ JEDINICI LIJEKA 20 mg/10 ml
English: In the historical cohort, 0 of 21 patients survived beyond 8 months of age (0% 12-month survival, 95% CI: 0% to
Croatian: U povijesnoj kohorti, 0 od 21 bolesnika preživjelo je više od 8 mjeseci (12-mjesečno preživljenje 0%, 95% CI: 0% do 16%).
English: There are no clinical data on the effects of sebelipase alfa on fertility.
Croatian: Nema kliničkih podataka o učincima sebelipaze alfa na plodnost.
English: If you or your child has an infusion reaction you or your child may be given additional medicines to treat or help prevent future reactions.
Croatian: Ako Vi ili Vaše dijete razvijete infuzijsku reakciju, možda će se Vama ili Vašem djetetu dati dodatni lijekovi za liječenje ili sprečavanje budućih reakcija.
English: Dose adjustments may be considered based on how well you or your child responds to treatment.
Croatian: Može se razmotriti prilagodba doze ovisno o Vašem odgovoru ili odgovoru Vašeg djeteta na liječenje.
English: The recommended dose is 1 mg per kg body weight once every other week through a drip into a vein.
Croatian: Preporučena doza je 1 mg po kg tjelesne težine jednom svaki drugi tjedan polaganim davanjem u venu (drip).
English: It also improves blood levels of fats, including elevated LDL (bad cholesterol) and triglycerides.
Croatian: Također se poboljšavaju razine masnoća u krvi, uključujući povišen LDL (loš kolesterol) i trigliceride.
English: KANUMA at doses up to 1 mg/kg once weekly resulted in improvements in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels and weight gain within the first several weeks of treatment.
Croatian: Liječenje KANUMOM u dozama do 1 mg/kg jednom tjedno imalo je za posljedicu poboljšanje razina alanin aminotransferaze (ALT) i aspartat aminotransferaze (AST) te dobitak na težini unutar prvih nekoliko tjedana liječenja.
English: 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.
Croatian: Ispitivanja kronične toksičnosti u mladih makaki majmuna nisu pokazala toksičnost pri dozama do 3 puta većima od preporučenih doza za dojenčad i 10 puta većima od preporučenih doza u odraslih/djece.
English: Study LAL-CL08: an open-label, Phase 2 study in infants with rapidly progressive LAL Deficiency to explore long-term safety and efficacy data.
Croatian: Ispitivanje LAL-CL08: otvoreno ispitivanje faze 2 u dojenčadi s brzo progresivnim nedostatkom LAL-a kako bi se prikupili podaci o dugoročnoj sigurnosti i djelotvornosti.
English: LAL deficiency is a genetic disease that leads to liver damage, high blood cholesterol, and other complications due to a build-up of certain types of fats (cholesteryl esters and triglycerides).
Croatian: Nedostatak LAL-a je genetska bolest koja dovodi do oštećenja jetre, visokih razina kolesterola u krvi i drugih komplikacija zbog nakupljanja određenih vrsta masnoća (kolesterolskih estera i triglicerida).
English: In LAL-CL03, 6 of 9 KANUMA-treated infants survived beyond 12 months (67% 12-month survival, 95% CI: 30% to 93%).
Croatian: U ispitivanju LAL-CL03, 6 od 9 dojenčadi liječene KANUMOM preživjelo je više od 12 mjeseci (12-mjesečno preživljenje 67%, 95% CI: 30% do 93%).
English: Risk Management Plan (RMP) 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.
Croatian: Nositelj odobrenja obavljat će dodatne farmakovigilancijske aktivnosti i intervencije, detaljno objašnjene u dogovorenom Planu upravljanja rizikom, a koji je opisan u Modulu 1.8.2 Odobrenja za stavljanje lijeka u promet, te svim sljedećim dogovorenim nadopunama Plana.
English: After dilution: Chemical and physical in-use stability has been demonstrated for up to 24 hours at 2°C to 8°C, or up to 12 hours below 25°C.
Croatian: Nakon razrjeđivanja: Dokazana je kemijska i fizikalna stabilnost lijeka u primjeni u trajanju do 24 sata pri temperaturi od 2 °C do 8 °C ili do 12 sati pri temperaturi ispod 25 °C.
English: Study LAL-CL02 - Children and Adults 1 mg/kg once every other week
Croatian: Ispitivanje LAL-CL02 - djeca i odrasli 1 mg/kg jednom svaki drugi tjedan 4-11 godina starosti n=24
English: Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.
Croatian: Unutar svake skupine učestalosti nuspojave su prikazane od ozbiljnijih prema manje ozbiljnima.
English: 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.
Croatian: Klinička slika nedostatka LAL-a u dojenčadi je medicinski hitno stanje s brzom progresijom bolesti tijekom nekoliko tjedana te obično završava smrtnim ishodom unutar prvih 6 mjeseci života.
English: Primary Endpoint Normalisation of ALT
Croatian: Primarni ishod Normalizacija ALT-a a
English: Sebelipase alfa is similar to the naturally occurring enzyme lysosomal acid lipase (LAL), which the body uses to breakdown fats.
Croatian: Sebelipaza alfa je slična prirodnom enzimu lizosomskoj kiseloj lipazi (LAL), koji tijelo koristi za razgradnju masnoća.
English: Intravenous use after dilution.
Croatian: Primjena u venu nakon razrjeđivanja.
English: A further dose escalation to 5 mg/kg once weekly was allowed.
Croatian: Bilo je dopušteno daljnje povećanje doze na 5 mg/kg jednom tjedno.
English: Patients received sebelipase alfa at 0.35 mg/kg once weekly for the first 2 weeks and then 1 mg/kg once weekly.
Croatian: Bolesnici su primali sebelipazu alfa u dozi od 0,35 mg/kg jednom tjedno tijekom prva 2 tjedna, a zatim 1 mg/kg jednom tjedno.
English: The following endpoints were assessed: normalisation of ALT, decrease in LDL-cholesterol, decrease in non-HDL-cholesterol, normalisation of AST, decrease in triglycerides, increase in HDLcholesterol, decrease in liver fat content assessed by multi-echo gradient echo magnetic resonance imaging (MEGE-MRI), and improvement in hepatic steatosis measured by morphometry.
Croatian: Procjenjivane su sljedeće ishodi: normalizacija ALT-a, snižavanje razine LDL kolesterola, snižavanje razine ne-HDL kolesterola, normalizacija AST-a, snižavanje razine triglicerida, porast razine HDL kolesterola, smanjenje količine masti u jetri procijenjeno pomoću MEGE-MR-a (engl. multi-echo gradient echo magnetic resonance imaging, MEGE-MRI) i poboljšanje jetrene steatoze mjereno morfometrijom.
English: In a separate open-label study (LAL-CL01/LAL-CL04) in adult patients with LAL deficiency, improvements in serum transaminase and lipid levels were sustained through the 104-week treatment period.
Croatian: U zasebnom otvorenom ispitivanju (LAL-CL01/LAL-CL04) u odraslih bolesnika s nedostatkom LALa, postignuta poboljšanja serumskih transaminaza i razina lipida održana su tijekom 104 tjedna liječenja.
English: Evaluated in patients with abnormal baseline values (n = 36 for KANUMA; n = 29 for placebo).
Croatian: Procijenjeno u bolesnika s abnormalnim početnim vrijednostima (n=36 za KANUMU; n=29 za placebo).
English: Based on clinical response, dose escalation to 3 mg/kg once weekly occurred as early as 1 month and up to 20 months after starting treatment at 1 mg/kg.
Croatian: Temeljeno na kliničkom odgovoru, povećanje doze na 3 mg/kg jednom tjedno uvedeno je već od 1 mjesec pa sve do 20 mjeseci nakon početka liječenja s 1 mg/kg.
English: Contents of the pack and other information
Croatian: Sadržaj pakiranja i druge informacije Što KANUMA sadrži
English: Pack sizes: 1 vial containing 10 ml of concentrate.
Croatian: Veličine pakiranja: 1 bočica s 10 ml koncentrata.
English: There are no data from the use of sebelipase alfa in pregnant women.
Croatian: Nema podataka o primjeni sebelipaze alfa u trudnica.
English: The pharmacokinetics of sebelipase alfa in children and adults were determined using a population pharmacokinetic analysis of 65 patients with LAL deficiency who received intravenous infusions of KANUMA at 1 mg/kg once every other week in LAL-CL02.
Croatian: Farmakokinetika sebelipaze alfa u djece i odraslih utvrđena je analizom populacijske farmakokinetike u 65 bolesnika s nedostatkom LAL-a koji su primali intravenske infuzije KANUME u dozi od 1 mg/kg jednom svaki drugi tjedan u ispitivanju LAL-CL02.
English: Paired liver biopsies at baseline and week 20 were available in a subset of patients (n = 26).
Croatian: Upareni uzorci biopsije jetre na početku i u 20. tjednu bili su dostupni za podskupinu bolesnika (n=26).
English: Due to the proteinaceous nature of the product, slight flocculation (e.g., thin translucent fibres) may be present in the vialed solution and is acceptable for use.
Croatian: Budući da je lijek proteinskog sastava, moguća je lagana flokulacija otopine u bočici (npr. tanka prozirna vlakna) i to je prihvatljivo za primjenu.
English: Reported as part of an adverse reaction in a single patient receiving KANUMA in LAL-CL02
Croatian: Zabilježeno kao dio nuspojave u jednog bolesnika koji je primao KANUMU u ispitivanju LAL-CL02
English: Anxietyc, insomnia
Croatian: Anksioznost c, nesanica
English: The other ingredients are trisodium citrate dihydrate (see section 2 under ‘KANUMA contains sodium'), citric acid monohydrate, human serum albumin, and water for injections.
Croatian: Drugi sastojci su trinatrijev citrat dihidrat (vidjeti dio 2 pod ‘KANUMA sadrži natrij'), citratna kiselina hidrat, ljudski serumski albumin i voda za injekcije.
English: Sebelipase alfa binds to cell surface receptors via glycans expressed on the protein and is subsequently internalized into lysosomes.
Croatian: Sebelipaza alfa se veže na površinske stanične receptore preko glikana eksprimiranih na proteinu te se zatim unosi u lizosome.
English: Table 5: Recommended infusion volumes (1 mg/kg dose)* Weight range (kg)
Croatian: Tablica 5: Preporučeni volumeni infuzije (doza od 1 mg/kg)* Raspon težine (kg)
English: LAL deficiency is a rare disease associated with significant morbidity and mortality, which affects individuals from infancy through adulthood.
Croatian: Nedostatak LAL-a je rijetka bolest povezana sa značajnim pobolom i smrtnošću koja zahvaća osobe od dojenačke do odrasle dobi.
English: KANUMA treatment should be supervised by a healthcare professional experienced in the management of patients with LAL deficiency, other metabolic disorders, or chronic liver diseases.
Croatian: Liječenje KANUMOM treba nadzirati zdravstveni radnik s iskustvom u liječenju bolesnika s nedostatkom LAL-a, drugim poremećajima metabolizma ili kroničnim bolestima jetre.
English: The educational material is aimed to encourage healthcare professionals to enrol patients in the prospective disease and clinical outcome registry of patients with Lysosomal Acid Lipase (LAL) Deficiency to monitor for efficacy and safety of Kanuma (LAL Deficiency Registry), with particular regard to hypersensitivity reactions, including anaphylaxis, and anti-drug antibodies (ADA) development impacting response to drug.
Croatian: Edukacijski materijal ima za cilj potaknuti zdravstvene radnike da uključe bolesnike u prospektivni registar bolesti i kliničkog ishoda bolesnika s nedostatkom lizosomske kisele lipaze (LAL-a) za praćenje djelotvornosti i sigurnosti Kanume (Registar nedostatka LAL-a), posebno u pogledu reakcija preosjetljivosti, uključujući anafilaksiju, te utjecaja razvoja protutijela na lijek (ADA) na odgovor na lijek.
English: Efficacy was assessed by comparing the survival experience of KANUMA-treated patients who survived past 12 months of age in LAL-CL03 with a historical cohort of untreated infants presenting with LAL deficiency with similar clinical characteristics.
Croatian: Djelotvornost je procijenjena usporedbom preživljenja bolesnika liječenih KANUMOM koji su doživjeli dob preko 12 mjeseci u ispitivanju LAL-CL03 s povijesnom kohortom neliječene dojenčadi s kliničkom slikom nedostatka LAL-a sa sličnim kliničkim karakteristikama.
English: Secondary Endpoints
Croatian: Sekundarni ishod
English: Because it is a recombinant human protein, sebelipase alfa is an unlikely candidate for cytochrome P450 mediated or other drug-drug interactions.
Croatian: Budući da je sebelipaza alfa rekombinantni ljudski protein, sudjelovanje u interakcijama posredovanim citokromom P450 ili u drugim interakcijama među lijekovima nije vjerojatno.
English: For patients who have signs and symptoms of the disease when they are infants, the recommended starting dose is 1 mg/kg once weekly.
Croatian: Za bolesnike koji imaju znakove i simptome bolesti u dojenačkoj dobi preporučuje se početna doza od 1 mg/kg jednom tjedno.