FrancophonIA/ELRC3.0 huggingface dataset:
English: In study 190201, 23 patients were treated for 48 weeks (1 patient withdrew after week 1 due to the inability to continue with study procedures).
Danish: I studie 190201 blev 23 patienter behandlet i 48 uger (1 patient stoppede efter uge 1 pga. manglende evne til at fortsætte med procedurerne i studiet).
English: Inspect the vials to ensure they are fully thawed.
Danish: Undersøg hætteglassene for at sikre, at de er helt optøede.
English: 1 year to < 2 years: 200 mg (first 4 doses), 300 mg (all other doses) ≥ 2 years: 300 mg
Danish: 1 år til < 2 år: 200 mg (første 4 doser), 300 mg (alle andre doser) ≥ 2 år: 300 mg
English: Each domain encompasses scores of 3 (grossly normal) to 0 (profoundly impaired), for a total possible score of 6, with unit decrements representing milestone events in the loss of previously attained functions of ambulation and speech.
Danish: Hvert domæne omfatter scorer på 3 (samlet set normal) til 0 (meget forringet) til en samlet mulig score på 6, med aftagende enheder, der repræsenterer delmålshændelser i tab af tidligere opnåede funktioner inden for mobilisering og tale.
English: There were no patients with advanced disease progression at treatment initiation who were included in clinical trials and no clinical data is available in children < 2 years.
Danish: Der var ingen patienter med fremskreden sygdomsprogression ved behandlingsstart inkluderet i kliniske forsøg, og der findes ingen kliniske data for børn < 2 år.
English: Table 3: CLN2 Clinical Rating Scale
Danish: Tabel 3: Klinisk rating-skala for CLN2
English: The observed treatment effect was considered clinically meaningful in light of the natural history of untreated CLN2 disease.
Danish: Den observerede behandlingseffekt blev anset for at være klinisk meningsfuld set i lyset af den naturligt historiske, ubehandlede CLN2-sygdom.
English: • Secure the components per institution standard of care.
Danish: • Fikser komponenterne ifølge institutionens standardprocedurer.
English: Brineura is compatible with disposable infusion components made of PVC, PVC (non-DEHP) polyethylene, polyethersulfone (PES), polypropylene (PP), and PTFE.
Danish: Brineura er forligeligt med engangsinfusionskomponenter fremstillet af PVC, PVC (ikke-DEHP) polyethylen, polyethersulfon (PES), polypropylen (PP) og PTFE.
English: Study 190-203 is an ongoing open label clinical study evaluating the safety and efficacy in patients from birth to 18 years of age.
Danish: Studie 190-203 er et igangværende, åbent, klinisk studie, der evaluerer sikkerheden og virkningen hos patienter fra fødslen til 18 år.
English: Will have obvious instability, and may have intermittent falls.
Danish: Udviser tydelig ustabilitet og kan falde periodisk.
English: If you or your child has a history of heart problems, your doctor or nurse will monitor your heart activity during each infusion.
Danish: Hvis du eller dit barn tidligere har haft problemer med hjertet, vil lægen eller sygeplejersken måle hjerteaktiviteten under hver infusion.
English: Motor
Danish: Motorisk
English: Domain Score
Danish: Domæne Score
English: In case of intracerebroventricular access device-related complications, refer to the manufacturer's labelling for further instruction.
Danish: Hvis der er intracerebroventrikulære adgangsudstyrsrelaterede komplikationer, henvises der til producentens mærkning angående yderligere anvisninger.
English: Brineura contains the active substance cerliponase alfa, which belongs to a group of medicines known as enzyme replacement therapies.
Danish: Brineura indeholder det aktive stof cerliponase alfa, som tilhører en gruppe af lægemidler, der kaldes enzymsubstitutionsbehandling.
English: Tmax*, hr
Danish: Tmaks*, t
English: frequency convention defined as: very common (≥1/10), common (≥1/100 to <1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000), not known (cannot be estimated from the available data).
Danish: MedDRA-grupperinger angivet nedenfor: Meget almindelig (≥1/10), almindelig (≥1/100 til <1/10), ikke almindelig (≥1/1.000 til <1/100), sjælden (≥1/10.000 til <1/1.000), meget sjælden (<1/10.000),
English: 4.2 Posology and method of administration
Danish: 4.2 Dosering og administrationsmetode
English: There are no data on the presence of cerliponase alfa in human milk, the effects of cerliponase alfa on the breastfed child, or the effects of cerliponase alfa on milk production.
Danish: Der er ingen data om tilstedeværelse af cerliponase alfa i human mælk, om cerliponase alfas virkning på det ammede barn eller om cerliponase alfas påvirkning af mælkeproduktionen.
English: No prominent ataxia, no pathologic falls.
Danish: Ingen fremtrædende ataksi, ingen patologiske fald.
English: Table 1: Dose and volume of Brineura
Danish: Tabel 1: Dosis og volumen af Brineura
English: Table 4: 0 to 6 Point Motor-Language CLN2 Clinical Rating Scale: Rate of Decline over 48 weeks (Intent to Treat (ITT) population)
Danish: Tabel 4: 0 til 6 point motorisk/sproglig klinisk rating-skala for CLN2: Forringelsesrate over 48 uger (Behandlingsintention (ITT)-population)
English: - If you or your child has had life-threatening allergic reactions to cerliponase alfa or any of the other ingredients of this medicine (listed in section 6), and the reactions continue to happen when cerliponse alfa is given again.
Danish: - hvis du eller dit barn har haft en livstruende allergisk reaktion på cerliponase alfa eller et af de øvrige indholdsstoffer i dette lægemiddel (anført i afsnit 6), og den allergiske reaktion sker igen, når cerliponase alfa indgives igen.
English: No fertility studies with cerliponase alfa have been conducted in animals or humans.
Danish: Der er ikke udført fertilitetsstudier med cerliponase alfa hos dyr eller mennesker.
English: Tell your doctor if you or your child is taking, has recently taken, or might take any other medicines.
Danish: Fortæl altid lægen, hvis du eller dit barn tager anden medicin, har gjort det for nylig, eller planlægger at tage anden medicin.
English: Cerliponase alfa is a proteolytic inactive proenzyme (zymogen) that is activated in the lysosome.
Danish: Cerliponase alfa er et proteolytisk inaktivt proenzym (zymogen), der aktiveres i lysosomet.
English: CLN2 patients with ventriculo-peritoneal shunts.
Danish: CLN2-patienter med ventriculoperitoneal shunt.
English: The complete infusion time, including Brineura and the required flushing solution, is approximately 2 to 4.5 hours, depending on the dose and volume administered.
Danish: Den samlede infusionstid, herunder Brineura og den påkrævede volumen skyllevæske, er ca. 2 til 4,5 time, afhængigt af administreret dosis og volumen.
English: It is not known whether Brineura can cause foetal harm when administered to a pregnant woman or can affect reproductive capacity.
Danish: Det er ukendt, om Brineura kan forårsage fosterskade, hvis det administreres til en gravid kvinde, eller om det kan påvirke forplantningsevnen.
English: Brineura has not been given to patients with advanced disease at the start of treatment or in children younger than 2 years of age.
Danish: Brineura er ikke blevet givet til patienter med fremskreden sygdom i starten af behandling eller til børn under 2 år gamle.
English: As a precautionary measure, appropriate medical support should be readily available when Brineura is administered.
Danish: Som forsigtighedsforanstaltning skal der være korrekt medicinsk støtte til rådighed, mens Brineura administreres.
English: Prior to the launch of BRINEURA in each Member State (MS), the Marketing Authorisation Holder (MAH) must agree about the content and format of the educational materials, including communication media, distribution modalities, and any other aspects of the programme, with the National Competent Authority (NCA).
Danish: Inden lanceringen af BRINEURA i hver medlemsstat skal indehaveren af markedsføringstilladelsen aftale indholdet og formatet af oplysningsmaterialerne, herunder kommunikationsmedie, distributionsmodaliteter og eventuelle øvrige aspekter af programmet, med de nationale kompetente myndigheder.
English: Your doctor may adjust you or your child's dose or the amount of time the medicine is given if the infusion is not tolerated, there is an allergic reaction or there is a possible increase of pressure in the brain.
Danish: Lægen kan justere din eller dit barns dosis eller den tid hvori lægemidlet gives, hvis infusionen ikke tolereres, der er en allergisk reaktion, eller der er en mulig stigning i trykket i hjernen.
English: The posology proposed in children below 2 years has been estimated based on brain mass.
Danish: Den foreslåede dosering for børn under 2 år er blevet anslået baseret på hjernemasse.
English: Your doctor will discuss whether Brineura treatment is right for you or your child.
Danish: Lægen vil tale med dig, om Brineura i behandlingen er rigtig for dig eller dit barn.
English: 6.7 (first 4 doses) 10 (subsequent doses)
Danish: 6,7 (første 4 doser) 10 (efterfølgende doser)
English: If immediate use is not possible, unopened vials of Brineura or flushing solution should be stored at 2-8°C and used within 24 hours.
Danish: Hvis øjeblikkelig anvendelse ikke er mulig, skal uåbnede hætteglas med Brineura eller skyllevæske opbevares ved 2-8 °C og anvendes inden 24 timer.
English: Continuation of long-term treatment should be subject to regular clinical evaluation whether the benefits are considered to outweigh the potential risks to individual patients.
Danish: Kontinuerlig langtidsbehandling skal kontrolleres regelmæssigt med klinisk evaluering for at vurdere, om de kliniske fordele lader til at opveje de potentielle risici for de individuelle patienter.
English: These adverse reactions were observed during or within 24 hours after completion of the Brineura infusion and did not interfere with treatment.
Danish: Disse bivirkninger blev observeret under eller inden for 24 timer efter afsluttet infusion af Brineura og forstyrrede ikke behandlingen.
English: There is limited experience in a few patients aged 2 years old.
Danish: Der er begrænset erfaring med få patienter, der er 2 år gamle.
English: The activated proteolytic enzyme (rhTPP1) cleaves tripeptides from the N-terminus of the target protein with no known substrate specificity.
Danish: Det aktiverede, proteolytiske enzym (rhTPP1) spalter tripeptider fra målproteinets N-terminal uden nogen kendt substratspecificitet.
English: Cerliponase alfa is a recombinant form of human tripeptidyl peptidase-1 (rhTPP1).
Danish: Cerliponase alfa er en rekombinant form af humant tripeptidyl peptidase-1 (rhTPP1).
English: Post-authorisation efficacy study (PAES): Study 190-203.
Danish: Virkningsundersøgelse efter tilladelse til markedsføring (PAES): Studie 190-203.
English: You or your child may get problems with the implanted device used during treatment with Brineura (see section 4 “Possible side effects”), including infection or a fault in the device.
Danish: Du eller dit barn kan få problemer med den implanterede anordning, der bruges under behandlingen med Brineura (se afsnit 4, ”Bivirkninger”), herunder infektion eller fejl ved anordningen.
English: This medicine works to slow the progression of the disease.
Danish: Lægemidlet sinker sygdomsudviklingen.
English: Symptoms resolved over time or with administration of antipyretics, antihistamines and/or glucocorticosteroids.
Danish: Symptomerne forsvandt med tiden eller efter administration af feberstillende midler, antihistaminer og/eller glukokortikosteroider.
English: What you need to know before you or your child is given Brineura You must not receive Brineura:
Danish: Det skal du vide, før du eller dit barn får Brineura Du må ikke få Brineura:
English: 200 (first 4 doses) 300 (subsequent doses)
Danish: 200 (første 4 doser) 300 (efterfølgende doser)
English: However, the results of these studies in dachshund dogs cannot reliably predict human safety, because the regimen of cerliponase alfa infusions was different and highly variable even within the same study due to difficulties with the indwelling catheter system and prominent hypersensitivity reactions.
Danish: Resultaterne af disse studier med gravhunde kan imidlertid ikke i pålidelig grad forudsige sikkerheden for mennesker, fordi infusionsregimet af cerliponase alfa var anderledes og varierede meget, selv inden for samme studie, på grund af vanskeligheder med katetre a demeure og udtalte overfølsomhedsreaktioner.
English: In order to further evaluate the treatment effectiveness as a delay in progression of CLN2 motor-language clinical sale and to further evaluate the safety and tolerability of cerliponase alfa, the MAH will submit the results of study 190-203 including at least 5 patients below the age of 2 years.
Danish: Med henblik på yderligere evaluering af den effektive virkning af behandlingen i form af udsættelse af progressionen af CLN2-relaterede motoriske og sproglige funktionstab og med henblik på yderligere evaluering af sikkerheden og tolerabiliteten af cerliponase alfa skal indehaveren af markedsføringstilladelsen indsende resultaterne af studie 190-203, som inkluderer mindst 5 patienter i alderen under 2 år.
English: The recommended dose of Brineura is based upon your or your child's age, and is given once every other week as follows:
Danish: Den anbefalede dosis Brineura fastlægges ud fra din eller dit barns alder og indgives af en læge eller sygeplejerske en gang hver anden uge efter denne doseringsplan:
English: 6 months to < 1 year: 150 mg
Danish: 6 måneder til < 1 år: 150 mg
English: Findings from studies 190-201 and 190-202 were compared with a natural history control group that included patients that satisfied the inclusion criteria for studies 190-201 and 190-202.
Danish: Fund fra studie 190-201 og 190-202 blev sammenlignet med en naturligt historisk kontrolgruppe, der omfattede patienter, som opfyldte inklusionskriterierne for studie 190-201 og 190-202.
English: Talk to your doctor or nurse immediately if you experience any of the following:
Danish: Fortæl det straks til lægen eller sygeplejersken, hvis du får en eller flere af følgende bivirkninger:
English: The intracerebroventricular access device must be implanted prior to the first infusion.
Danish: Udstyret til intracerebroventrikulær adgang skal implanteres inden den første infusion.
English: These naturally occurring particles are cerliponase alfa.
Danish: Disse naturligt forekommende partikler er cerliponase alfa.
English: Anti-drug antibodies (ADAs) were detected in both serum and CSF in 79% and 21%, respectively, of patients treated with cerliponase alfa for up to 107 weeks.
Danish: Der blev detekteret antistoffer mod lægemidlet (ADA) i både serum og CSV hos hhv. 79 % og 21 % af patienter, der blev behandlet med cerliponase alfa i op til 107 uger.
English: Treatment of Brineura was initiated in children 2 to 8 years of age in clinical studies.
Danish: Behandling af Brineura blev påbegyndt hos børn 2 til 8 år gamle i kliniske forsøg.
English: Renal elimination of cerliponase alfa is considered a minor pathway for clearance.
Danish: Renal udskillelse af cerliponase alfa anses som en mindre clearance-vej.
English: No patients had to have their treatment discontinued due to adverse events.
Danish: Ingen patienter fik seponeret behandlingen pga. bivirkninger.
English: If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user.
Danish: Hvis de ikke anvendes straks, er opbevaringstider og -forhold før anvendelse brugerens ansvar.
English: • Do not deliver as a bolus or manually.
Danish: • Lægemidlet må ikke administreres som bolus eller administreres manuelt.
English: Pharmacotherapeutic group: other alimentary tract and metabolism products, enzymes, ATC code: A16AB17.
Danish: Farmakoterapeutisk klassifikation: andre fordøjelseskanal- og metabolismeprodukter, enzymer, ATCkode: A16AB17..
English: After the medicine has been given, a shorter infusion of a solution is given to flush Brineura out of the infusion equipment so that the full dose reaches the brain.
Danish: Når lægemidlet er indgivet, følger en kortere infusion med en væske for at skylle Brineura ud af infusionsudstyret, så hele dosen når hjernen.
English: Brineura should be given to a pregnant woman only if clearly needed.
Danish: Brineura bør kun gives til en gravid kvinde, hvis det er strengt nødvendigt.
English: Brineura must be administered using aseptic technique to reduce the risk of infection.
Danish: Brineura skal administreres vha. aseptisk teknik for at mindske infektionsrisikoen.
English: • A 0.2 µm inline filter is required.
Danish: • Det er nødvendigt at bruge et 0,2 µm inline-filter.
English: Your doctor may decide to continue treatment once the device infection or problems are resolved.
Danish: Lægen kan beslutte at fortsætte behandlingen, når betændelsen eller problemerne med anordningen er forsvundet.
English: The inline filter may be integral to the infusion set.
Danish: Inline-filteret kan være en indbygget del af infusionssættet.
English: Cerliponase alfa is a protein and is expected to be metabolically degraded through peptide hydrolysis.
Danish: Cerliponase alfa er et protein, og det forventes at nedbrydes metabolisk gennem hydrolyse af peptider.
English: Posology was based upon analysis of differences in brain mass values for children less than 3 years of age.
Danish: Dosering blev baseret på analyse af forskelle i hjernemasseværdier for børn under 3 år.
English: Cerliponase alfa is taken up by target cells and translocated to the lysosomes through the Cation Independent Mannose-6-Phosphate Receptor (CI-MPR, also known as M6P/IGF2 receptor).
Danish: Cerliponase alfa optages af målcellerne og translokeres til lysosomerne via den kationsuafhængige mannose-6-fosfat-receptor (CIMPR, også kaldet M6P/IGF2-receptor).
English: IRELAND
Danish: Irland
English: Results from the natural history control group demonstrate CLN2 disease is a rapidly progressive neurodegenerative disease with predictable decline in motor and language function with an estimated mean rate of decline in the CLN2 score of 2 points per 48 weeks.
Danish: Resultater fra den naturligt historiske kontrolgruppe viser, at CLN2 er en hurtigt progredierende neurodegenerativ sygdom med forudsigelig forringelse af den motoriske funktion og sprogfunktionen med en estimeret gennemsnitlig forringelsesrate i CLN2-scoren på 2 point på 48 uger.
English: The flushing solution may contain particles that dissolve when the vial is fully thawed.
Danish: Skyllevæsken kan indeholde partikler, der opløses, når hætteglasset er helt optøet.
English: Consultation with a neurosurgeon may be needed to confirm the integrity of the device.
Danish: Det kan være nødvendigt at konsultere en neurokirurg for at bekræfte, at udstyret er intakt.
English: Caution should be taken in patients prone to complications from intracerebroventricular medicinal product administration, including patients with obstructive hydrocephalus.
Danish: Der skal udvises forsigtighed hos patienter med tendens til komplikationer under intracerebroventrikulær administration af lægemidler, herunder patienter med obstruktiv hydrocephalus.
English: These particles are removed via the 0.2 μm inline filter without having a detectable effect on the purity or strength of Brineura.
Danish: Partiklerne fjernes i det 0,2 μm inline-filter, uden det har nogen detekterbar virkning på Brineuras renhed eller styrke.
English: Drug-specific neutralizing antibodies (NAb) capable of inhibiting receptor-mediated cellular uptake of cerliponase alfa were not detected in the CSF.
Danish: Lægemiddelspecifikke, neutraliserende antistoffer (NAb), der kan hæmme receptormedieret cellulært optag af cerliponase alfa, blev ikke detekteret i CSV.
English: People with CLN2 disease do not have any enzyme called TPP1 or they have too little of it and this causes a build-up of substances called lysosomal storage materials.
Danish: Mennesker med CLN2-sygdom har ikke et enzym, der hedder TPP1, eller de har for lidt af det, og det forårsager en ophobning af stoffer, der hedder lysosomale aflejringer.
English: Your doctor may give you or your child medicines, such as antipyretics to reduce fever or antihistamines to treat allergic reactions before each treatment with Brineura to reduce side effects that can occur during or shortly after treatment.
Danish: Lægen giver muligvis dig eller dit barn anden medicin, såsom feberstillende midler mod feber eller antihistaminer mod allergiske reaktioner, inden hver behandling med Brineura for at mindske de bivirkninger, der kan opstå under eller kort efter behandlingen.
English: Electrocardiogram (ECG) monitoring during infusion should be performed in patients with a history of bradycardia, conduction disorder, or with structural heart disease, as some patients with CLN2 disease may develop conduction disorders or heart disease.
Danish: Der bør udføres monitorering med elektrokardiogram (EKG) under infusionen hos patienter med tidligere bradykardi, ledningsforstyrrelser eller strukturel hjertesygdom, da visse patienter med CLN2sygdom kan udvikle ledningsforstyrrelser eller hjertesygdom.
English: In cardiac normal patients, regular 12-lead ECG evaluations should be performed every 6 months.
Danish: Hos patienter med normal hjertefunktion skal der tages almindeligt 12-aflednings-EKG hver 6. måned.
English: Do not thaw or warm vials any other way.
Danish: Hætteglassene må ikke optøs eller opvarmes på nogen anden måde.
English: The adverse reactions described in this section were evaluated in 24 patients with CLN2 disease who received at least one dose of Brineura in clinical studies of up to 141 weeks.
Danish: De bivirkninger, der er beskrevet i dette afsnit, blev evalueret hos 24 patienter med CLN2-sygdom, som fik mindst én dosis Brineura i kliniske studier, der varede op til 141 uger.
English: - The active substance is cerliponase alfa.
Danish: - Aktivt stof: Cerliponase alfa.
English: GENERAL CLASSIFICATION FOR SUPPLY
Danish: GENERELKLASSIFIKATION FOR UDLEVERING
English: Brineura has a green flip-off cap and flushing solution has a yellow flip-off cap.
Danish: Brineura har en grøn flip-off-hætte, og skyllevæsken har en gul flip-off-hætte.
English: • Two single-use hypodermic syringe needles, (21 G, 25.4 mm).
Danish: • To hypodermiske sprøjtekanyler til engangsbrug (21 G, 25,4 mm).
English: Animal reproduction studies have not been conducted using Brineura.
Danish: Der er ikke udført reproduktionsstudier med dyr, der får Brineura.
English: If the infusion is interrupted due to a hypersensitivity reaction, it should be restarted at approximately onehalf the initial infusion rate at which the hypersensitivity reaction occurred.
Danish: Hvis infusionen afbrydes pga. en overfølsomhedsreaktion, skal den genstartes ved en ca. halvt så hurtig infusionshastighed, som hastigheden var på det tidspunkt, hvor overfølsomhedsreaktionen indtraf.
English: Very common Common Common Not known
Danish: Meget almindelig Almindelig Almindelig Ikke kendt
English: Withdraw 0.5 ml to 1 ml of CSF to check patency of the intracerebroventricular access device.
Danish: Træk 0,5 ml til 1 ml CSV ud for at kontrollere åbenhed i udstyret til intracerebroventrikulær adgang.
English: This medicine replaces the missing enzyme, TPP1, which minimises the build-up of the lysosomal storage materials.
Danish: Dette lægemiddel erstatter det manglende enzym, TPP1, hvilket mindsker ophobningen af de lysosomale aflejringer.
English: Severe (Common Terminology Criteria for Adverse Events (CTCAE) grade 3) hypersensitivity reactions occurred in three patients and no patients discontinued treatment.
Danish: Svære overfølsomhedsreaktioner (Common Terminology Criteria for Adverse EventsCTCAE-grad 3) forekom hos tre patienter, og ingen patienter fik seponeret behandlingen.
English: The inline filter should be placed as close as practically possible to the port needle.
Danish: Inline-filteret skal sidde så tæt som praktisk muligt på portkanylen.