FrancophonIA/ELRC3.0 huggingface dataset:
English: Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.
Danish: Inden for hver hyppighedsgruppe angives bivirkninger efter aftagende alvorlighed.
English: The pH of the reconstituted solution is approximately 6.2.
Danish: Den rekonstituerede opløsnings pH-værdi er ca. 6.2.
English: The co-primary efficacy outcome assessments were distance walked (meters) in 6 minutes (6-Minute Walk Test, 6MWT) and FVC (Forced Vital Capacity) % predicted in the sitting position.
Danish: Undersøgelserne af den ekstra primære effektparameter omfattede gangdistance (meter) på 6 minutter (6-minute walk test, 6MWT) og FVC (forced vital capacity) % forventet i siddende stilling.
English: In the above described study in 69 late-onset patients in the Netherlands, Myozyme showed an improvement in muscle strength.
Danish: I det ovennævnte beskrevne hollandske studie hos 69 patienter med sent debuterende Pompes sygdom viste Myozyme en forbedring af muskelstyrken.
English: Subjects in the untreated historical cohort were born in 1993 or later.
Danish: Forsøgspersoner i den ubehandlede, historiske gruppe var født i 1993 eller senere.
English: Infusion Associated Reactions
Danish: Infusionsrelaterede reaktioner
English: Common: may affect up to 1 in 10 people
Danish: Almindelig (kan forekomme hos op til 1 ud af 10 personer):
English: The late-onset form of Pompe disease manifests during infancy, childhood, adolescence or even adulthood and is much less rapidly progressive than the infantile-onset form.
Danish: Den sent debuterende form af Pompes sygdom manifesterer sig i løbet af spædbørnsalderen, barndommen, ungdommen eller endda voksenalderen og er langt mindre hastigt progredierende end formen med debut i spædbørnsalderen.
English: AUC∞ and Cmax were similar at week 0, 12 and 52 visits indicating alglucosidase alfa pharmacokinetics were not timedependent (Table 5).
Danish: AUC og Cmax var identiske ved besøgene i uge 0, 12 og 52, hvilket tyder på, at farmakokinetikken for alglucosidase alfa ikke var tidsafhængig (Tabel 5).
English: A retrospective chart review at Duke Center identified 21 CRIM-negative IOPD patients of which 19 patients were ERT naïve at the time of ITI initiation.
Danish: En retrospektiv journalgennemgang ved Duke Center identificerede 21 CRIM-negative IOPD-patienter, hvoraf 19 patienter var enzymerstatningsterapi-naive, da ITI initieredes.
English: However, there was no apparent association between inhibition of uptake and the co-primary efficacy endpoints (see section 4.4).
Danish: Der var dog ingen tilsyneladende sammenhæng mellem hæmningen af optagelsen og de ekstra primære effektendepunkter (se pkt.
English: Alglucosidase alfa may be excreted in breast milk.
Danish: Alglucosidase alfa kan udskilles i modermælken.
English: Mean plasma elimination half-life (t1/2) was 2.75 hours for the two dose groups.
Danish: Gennemsnitlig plasmahalveringstid (t1/2) var 2,75 timer for de to dosisgrupper.
English: • Difficulty in breathing (including shortness of breath)
Danish: • Problemer med at trække vejret (herunder stakåndethed)
English: Cardiac arrest Bradycardia Tachycardia Cyanosis
Danish: Hjertestop Bradykardi Takykardi Cyanose
English: Subjects in the untreated historical cohort were born in 1995 or later.
Danish: Forsøgspersoner i den ubehandlede, historiske gruppe var født i 1995 eller senere.
English: Table 5: Alglucosidase alfa pharmacokinetics after a single dose and after 12 and 52 weeks of therapy
Danish: Tabel 5: Farmakokinetikken for alglucosidase alfa efter en enkelt dosis og efter 12 og 52 ugers behandling
English: • Pain or local reaction at the site of the drip
Danish: • Smerte eller lokal reaktion på stedet, hvor droppet indføres
English: Tachycardia common Cyanosis not known
Danish: Takykardi almindelig Cyanose ikke kendt
English: It is therefore recommended to perform periodic urinalysis among patients with high IgG antibody titres.
Danish: Det anbefales derfor regelmæssig at undersøge urinprøver hos patienter med høje IgG-antistoftitre.
English: Patients with Pompe disease are at increased risk of respiratory infections due to the progressive effects of the disease on the respiratory muscles.
Danish: Patienter med Pompes sygdom har øget risiko for luftvejsinfektioner på grund af sygdommens progressive virkninger på åndedrætsmusklerne.
English: Any unused product or waste material should be disposed of in accordance with local requirements.
Danish: Eventuelt ubrugt produkt eller affaldsmateriale skal bortskaffes i henhold til lokale krav.
English: In the untreated historical cohort 5 of 47 patients (10.6%) for whom data were available, were alive at age 30 months (2.5 years).
Danish: I den ubehandlede, historiske gruppe var 5 af 47 patienter (10,6 %) med tilgængelige data i live ved alderen 30 måneder (2,5 år).
English: Instructions for use - reconstitution, dilution and administration
Danish: Anvisninger vedrørende anvendelsen - rekonstituering, fortynding og indgivelse
English: This should then be further diluted as follows: Slowly withdraw the reconstituted solution from each vial until the volume for the patient's dose is obtained.
Danish: Denne skal derefter fortyndes yderligere som følger: Udtag langsomt den rekonstituerede opløsning fra hvert hætteglas, til mængden til patientens dosis er udtaget.
English: • Cold extremities (e.g. hands, feet)
Danish: • Kolde lemmer (f.eks. hænder og fødder)
English: You should not be given Myozyme during pregnancy unless clearly necessary.
Danish: Der bør ikke gives Myozyme under graviditet, medmindre det er klart nødvendigt.
English: 50 mg of powder in a vial (Type 1 glass) with a stopper (siliconised butyl) and a seal (aluminium) with a flip-off cap (plastic).
Danish: 50 mg pulver i et hætteglas (Type 1 glas) med en prop (silikonebelagt butyl) og en forsegling (aluminium) med vippehætte (plast).
English: Analyses of efficacy did not reveal meaningful differences between the 2 dose groups with respect to survival, invasive ventilator-free survival, any ventilator-free survival, decrease in LVM, gains in growth parameters and acquisition of motor milestones.
Danish: Analyser af effekt afslørede ingen forskelle af betydning mellem de 2 dosisgrupper med hensyn til overlevelse, overlevelse fri for invasiv ventilations-/respiratorbehandling, overlevelse uden nogen form for ventilationsbehandling, fald i LVM, forøgning i vækstparametre og opnåelse af motoriske milepæle.
English: • Swelling around the eyes
Danish: • Hævelse omkring øjnene
English: Better treatment responses were observed in younger patients with less advanced disease who received prophylactic ITI before development of HSAT, which suggests that early initiation of ITI can result in improved clinical outcomes.
Danish: Der blev observeret bedre behandlingsrespons hos yngre patienter med mindre fremskreden sygdom, som fik profylaktisk ITI, før de udviklede HSAT, hvilket tyder på, at tidlig initiering af ITI kan medføre forbedrede kliniske resultater.
English: 4 Additional adverse reactions from post-marketing, expanded access programs and non-controlled clinical trials.
Danish: 4 Yderligere bivirkninger, der er indberettet efter markedsføring, fra efterfølgende kliniske programmer og fra ikkekontrollerede kliniske undersøgelser.
English: Pre-treatment Baseline
Danish: Baseline inden behandling
English: Additional efficacy data showed that of 16 patients who were free of invasive-ventilator support at baseline, 7 remained so after 104 weeks of treatment.
Danish: Yderligere effektdata viste, at hos 16 patienter, der ikke modtog invasiv ventilationsbehandling ved baseline, var dette fortsat tilfældet for 7 patienter efter 104 ugers behandling.
English: Use aseptic technique • Reconstitution
Danish: Anvend aseptisk teknik • Rekonstituering
English: Patients should be monitored for signs and symptoms of systemic immune-mediated reactions involving skin and other organs while receiving alglucosidase alfa.
Danish: Patienter bør overvåges for tegn og symptomer på systemiske immunmedierede reaktioner, som involverer hud og andre organer under behandling med alglucosidase alfa.
English: • Sudden constriction of bronchi restricting air going in and out the lungs (bronchospasm)
Danish: • Pludselig sammentrækning af bronkier, hvilket indskrænker luftpassagen ind og ud af lungerne
English: After a minimum of 52 weeks, 16 of these 18 patients were enrolled in an extension study to receive continued treatment at the same dose for a total duration of up to three years (150 weeks).
Danish: Efter mindst 52 uger blev 16 af disse 18 patienter rekrutteret til et udvidet studie og fik fortsat behandling med samme dosis med en samlet varighed på op til tre år (150 uger).
English: After 52 weeks of treatment, all 18 patients treated with Myozyme were alive and 15 of these 18 patients were alive and free of invasive ventilatory support whereas 1 of 42 patients in the untreated historical cohort was alive at 18 months of age.
Danish: Efter 52 ugers behandling var alle 18 patienter, der blev behandlet med Myozome, i live og 15 ud af 18 patienter i live og fri for invasiv ventilationsbehandling, hvorimod 1 af 42 patienter i den ubehandlede, historiske gruppe var i live ved 18 måneders alderen.
English: After the first year (64 up to 130 weeks) of treatment LVM further decreased in 8 patients.
Danish: Efter det første år (64 til 130 uger) med behandling fald LVM yderligere hos 8 patienter.
English: However, chemical and physical in-use stability has been demonstrated for 24 hours at 2 to 8°C when stored under protection from light.
Danish: Kemisk og fysisk stabilitet efter fortynding er dog påvist i 24 timer ved 2 til 8°C ved opbevaring beskyttet mod lys.
English: After 104 weeks of treatment, 14 of 21 patients (66.7%) were alive and 1 patient was alive but had discontinued from the study.
Danish: Efter 104 ugers behandling var 14 af 21 patienter i live (66,7 %), og 1 patient var i live, men var trådt ud af studiet.
English: After reconstitution, it is recommended to promptly dilute the vials (see below).
Danish: Efter rekonstituering anbefales det straks at fortynde hætteglassene (se nedenfor).
English: Table 2: Results for endpoint survival using the Cox regression model
Danish: Tabel 2: Resultater for endepunkt overlevelse ved hjælp af Cox-regressionsmodel
English: Myozyme is given through a drip into a vein (by intravenous infusion).
Danish: Myozyme gives gennem et drop i en vene (ved intravenøs infusion).
English: Patients treated with Myozyme demonstrated prolonged survival as compared to survival in an untreated historical cohort (see Table 2).
Danish: Patienter, der blev behandlet med Myozyme, udviste længerevarende overlevelse sammenlignet med overlevelsen i den ubehandlede historiske gruppe (se Tabel 2).
English: Reduction of the infusion rate, temporary interruption of the infusion, or pre-treatment, generally with oral antihistamine and/or antipyretics and/or corticosteroids, has effectively managed most reactions.
Danish: Reduktion af infusionshastigheden, midlertidig afbrydelse af infusionen eller forudgående behandling, sædvanligvis med oral antihistamin og/eller antipyretika, har haft god virkning på de fleste reaktioner.
English: Myozyme is used to treat adults, children and adolescents of all ages who have a confirmed diagnosis of Pompe disease.
Danish: Myozyme bruges til behandling af voksne, børn og unge i alle aldre, som har en bekræftet diagnose på Pompes sygdom.
English: The risks and benefits of re-administering alglucosidase alfa following an immune-mediated reaction should be considered.
Danish: Risici og fordele ved at genoptage behandling med alglucosidase alfa efter en immunmedieret reaktion skal overvejes.
English: Life threatening reactions, including very severe generalised allergic reactions and anaphylactic shock, have been reported in some patients.
Danish: Der er indberettet livstruende reaktioner, herunder meget alvorlige generelle, allergiske reaktioner og anafylaktisk shock, hos nogle patienter.
English: However, the invasive ventilator-free survival was not recorded in the untreated historical cohort and a comparison of this endpoint is not possible.
Danish: Andelen af overlevende patienter uden respiratorbehandling blev dog ikke registreret i den ubehandlede, historiske gruppe, og en sammenligning af dette endepunkt er ikke mulig.
English: Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from infusion associated reactions.
Danish: Patienter med fremskreden Pompes sygdom kan have hæmmet hjerte- eller åndedrætsfunktion, hvilket kan prædisponere dem for en højere risiko for svære komplikationer i forbindelse med infusionsrelaterede reaktioner.
English: Hypertension Hypotension Vasoconstriction Pallor
Danish: Hypertension Hypotension Vasokonstriktion Bleghed
English: Of the 3 patients with significant pulmonary involvement at screening/baseline (percentage predicted forced vital capacity in the sitting position ranging from 5867%), two demonstrated clinically meaningful improvements in FVC (+11.5% and +16.0%) in the sitting position by Week 26.
Danish: Af de 3 patienter med signifikant pulmonal påvirkning ved screening/baseline (procenten for ”predicted forced vital capacity” (FVC) i siddende stilling varierede fra 58 - 67 %), udviste 2 klinisk betydelige forbedringer i FVC (+11,5 % og +16,0 %) i siddende stilling ved uge 26.
English: There is no experience with overdose of Myozyme.
Danish: Der er ingen erfaring med overdosering af Myozyme.
English: Note: Results are from a Cox proportional hazards regression analysis which includes treatment as a time-varying covariate, and also includes age of diagnosis and age at symptom onset.
Danish: Bemærk: Resultaterne er fra en Cox proportionel risiko regressions analyse, der inkluderer behandling som en tidsvarierende kovariat samt alder ved diagnose og alder ved symptomdebut.
English: If you experience a reaction like this, you should tell your doctor immediately.
Danish: De oplever en sådan reaktion, skal De omgående fortælle det til Deres læge.
English: Pompe disease belongs to the lysosomal storage disorders as it is caused by a deficiency of a naturally-occurring lysosomal hydrolase, acid αglucosidase (GAA) that degrades lysosomal glycogen to glucose.
Danish: Pompes sygdom tilhører de lysosomale aflejringssygdomme, idet den skyldes en mangel på en naturligt forekommende lysosomal hydrolase, sur α-glukosidase (GAA), der nedbryder lysosomal glykogen til glukose.
English: If you are treated with Myozyme, you may experience an infusion-associated reaction while you are being given the medicine or during the hours following the infusion.
Danish: Hvis De bliver behandlet med Myozyme, kan De eventuelt opleve en såkaldt infusionsrelateret reaktion, når De får medicinen eller i timerne efter infusionen.
English: Mean plasma clearance (CL) was 21.4 ml/h/kg and mean volume of distribution at steady state (Vss) was 66.2 ml/kg for both dose groups with small between-subject variability of 15% and 11%, respectively.
Danish: Gennemsnitlig plasmaclearance (CL) var 21,4 ml/t/kg og gennemsnitlig distributionsvolumen ved steady state (Vss) var 66,2 ml/kg for begge dosisgrupper med lille variation mellem patienterne på henholdsvis 15 % og 11 %.
English: People with Pompe disease have low levels of an enzyme called alpha-glucosidase.
Danish: Personer med Pompes sygdom har lave niveauer af et enzym, der kaldes for alfa-glukosidase.
English: In 12 patients with available data the AUC∞ and Cmax were approximately equivalent to those observed for the 20 mg/kg dose group in the pivotal trial.
Danish: Hos 12 patienter med tilgængelige data svarede AUC∞ og Cmax ca. til dem, der blev observeret for 20 mg/kg dosisgruppen i pivotalundersøgelsen.
English: Recurrent reactions consisting of flu-like illness or a combination of events such as fever, chills, myalgia, arthralgia, pain, or fatigue occurring post-infusion and lasting usually for a few days, have been observed in some patients treated with alglucosidase alfa.
Danish: Tilbagevendende reaktioner i form af af influenza-lignende sygdom eller en kombination af reaktioner som f.eks. feber, kulderystelser, myalgi, artralgi, smerter eller træthed, som forekommer efter infusionen og normalt varer nogle få dage, er blevet observeret hos nogle patienter behandlet med alglucosidase alfa.
English: The safety and efficacy of Myozyme in patients with renal or hepatic impairment have not been evaluated and no specific dose regimen can be recommended for these patients.
Danish: Myozymes sikkerhed og virkning hos patienter med nedsat nyre- eller leverfunktion er ikke vurderet, og der kan ikke gives nogen anbefalinger vedrørende dosering til sådanne patienter.
English: Due to the proteinaceous nature of the product, particle formation may occur in the reconstituted solution and final infusion bags.
Danish: På grund af produktets proteinagtige beskaffenhed kan der opstå partikeldannelse i den rekonstituerede opløsning og de endelige infusionsposer.
English: Myozyme treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases.
Danish: Behandling med Myozyme skal overvåges af en læge med erfaring i behandling af patienter med Pompes sygdom eller andre nedarvede stofskiftesygdomme eller neuromuskulære sygdomme.
English: There is no experience of the use of Myozyme in pregnant women.
Danish: Der er ingen erfaringer med brug af Myozyme til gravide.
English: After 52 weeks of treatment, 16 of 21 patients (76.2%) treated with Myozyme were alive.
Danish: Efter 52 ugers behandling var 16 af 21 patienter i live (76,2 %), som blev behandlet med Myozyme.
English: However, muscle function only improved in wheelchair independent patients and in those with less pronounced muscle weakness.
Danish: Muskelstyrken blev dog kun forbedret hos patienter, der ikke var afhængige af kørestol, og hos patienter med mindre udtalt muskelsvaghed.
English: Survival
Danish: Overlevelse
English: Careful consideration should be given to the patient's clinical status prior to administration of Myozyme.
Danish: Patientens kliniske status skal overvejes omhyggeligt før indgivelse af Myozyme.
English: 3 Reactions reported more frequently in the placebo group than in the Myozyme group in late-onset patients.
Danish: 3 Bivirkninger, der er indberettet hyppigere i placebogruppen end i Myozyme-gruppen med patienter med sen debut.
English: Remove airspace within the infusion bag.
Danish: Fjern luftrummet i infusionsposen.
English: The clinical presentation of Pompe disease can be described as a spectrum of disease which ranges from a rapidly-progressing infantile-onset form (onset of symptoms of Pompe disease typically within the first year of life and a very short expected life-span) to a less rapidly-progressing late-onset form.
Danish: Den kliniske præsentation af Pompes sygdom kan beskrives som et sygdomsspektrum, der varierer fra en hurtigt progredierende form, der debuterer i spædbørnsalderen (typisk start på symptomer på Pompes sygdom inden for første leveår og en meget kort forventet levetid) til en mindre hurtigt progredierende form med sen debut.
English: Pharmacotherapeutic group: Other alimentary tract and metabolism products, enzymes.
Danish: Farmakoterapeutisk gruppe: Andre midler til fordøjelsesorganer og stofskifte, enzymer.
English: Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency).
Danish: Myozyme er indiceret til langtids-enzymerstatningsterapi hos patienter med en bekræftet diagnose på Pompes sygdom (sur α-glukosidase-mangel).
English: In clinical studies, the majority of patients developed IgG antibodies to alglucosidase alfa typically within 3 months of treatment.
Danish: I kliniske studier udviklede flertallet af patienterne IgG-antistoffer mod alglucosidase alfa, typisk i løbet af de første 3 behandlingsmåneder.
English: In two additional open-label clinical trials with Myozyme with a follow-up of 24 months, ten patients with severe late-onset Pompe disease (moderate to severe motor impairment and assisted ventilation) showed a variable response on measures of motor and respiratory functions, mostly in the form of a modest improvement (AGLU03105, AGLU04107).
Danish: I to andre open-label kliniske undersøgelser med Myozyme med 24 måneders opfølgning udviste ti patienter med sent debuterende Pompes sygdom (moderat til svær motorisk svækkelse og assisteret ventilation) en variabel respons på målinger af motorisk funktion og lungefunktion, hovedsagelig i form af beskedne forbedringer (AGLU03105, AGLU04107).
English: Adverse reactions reported in at least 2 patients are listed in Table 1.
Danish: Bivirkninger, der blev indberettet fra mindst 2 patienter, er angivet i Tabel 1.
English: Patients with infantile-onset Pompe disease who demonstrated motor gains, had greater preservation of motor function and lower glycogen content in the quadriceps muscle at baseline.
Danish: Patienter med Pompes sygdom med debut i spædbørnsalderen, der udviste motorisk forbedring, havde større opretholdelse af motorisk funktion og lavere glykogenindhold i quadriceps-musklen ved baseline.
English: Nervous system disorders common Tremor
Danish: Nervesystemet almindelig Tremor
English: If you experience severe ulcerative lesions of your skin, please inform your doctor.
Danish: Hvis De oplever alvorlige sår på huden, skal De fortælle det til Deres læge.
English: After 104 weeks of treatment, all 16 patients who enrolled in the extension study were alive and 10 of these 16 patients were free of invasive ventilatory support.
Danish: Efter 104 ugers behandling var alle 16 patienter, som blev rekrutteret til det forlængede studie, i live, og 10 ud af disse 16 patienter var fri for invasiv ventilationsbehandling.
English: The recommended dosage of Myozyme is 20 mg per kg of body weight.
Danish: Den anbefalede dosis af Myozyme er 20 mg pr. kg kropsvægt.
English: Periorbital edema Livedo reticularis Lacrimation increased Rash
Danish: Periorbital ødem Livedo reticularis Forøget tåreflod Udslæt
English: Hypersensitivity/Anaphylactic reactions
Danish: Overfølsomhed/anafylaktiske reaktioner
English: In these patients renal biopsy showed immune complex deposition.
Danish: Hos disse patienter viste nyrebiopsi immunkompleksaflejring.
English: This should then be further diluted as follows: Slowly withdraw the reconstituted solution from each vial until the volume for the patient's dose is obtained using a syringe with a needle diameter not larger than 20 gauge.
Danish: Denne skal så fortyndes yderligere på følgende måde: Udtag langsomst den rekonstituerede opløsning fra hvert hætteglas, til mængden til patientens dosis er udtaget ved hjælp af en sprøjte med en nålediameter, der ikke overstiger 20 gauge.
English: Therefore, a 0.2 micron low protein binding in-line filter should be used for administration.
Danish: Derfor bør et 0,2 mikron in-line-filter med lav proteinbinding anvendes ved indgivelsen.
English: In a retrospective natural history study in patients with infantile-onset Pompe disease (n=168), the median age at onset of symptoms was 2.0 months and the median age of death was 9.0 months.
Danish: I et retrospektiv observationsstudie af patienter med Pompes sygdom med debut i spædsbørnsalderen (n=168) var den mediane alder ved symptomernes debut 2,0 måneder og den mediane alder ved død 9,0 måneder.
English: After reconstitution it is a clear, colourless to pale yellow solution, which may contain particles.
Danish: Efter rekonstituering er det en klar, farveløs til lysegul opløsning, der kan indeholde partikler.
English: System Organ Class
Danish: Systemorganklasse Hyppighed
English: Oxygen saturation decreased common Heart rate increased Blood pressure increased
Danish: Reduceret iltmætning almindelig Forhøjet hjertefrekvens Forhøjet blodtryk