natgillin/translations-raw huggingface dataset:
After the first year (64 up to 130 weeks) of treatment LVM further decreased in 8 patients.
Po prvem letu (64 do 130 tednov) zdravljenja se je vrednost LVM še dodatno znižala pri 8 bolnikih.
The cause of a poor clinical outcome and of developing high and sustained IgG antibody titres is thought to be multi-factorial.
Domnevajo, da je vzrok za nezadovoljiv klinični izid ter za razvoj visokih in vzdrževanih titrov protiteles IgG skupek več faktorjev.
• Nephrotic Syndrome: swelling of lower limbs, generalized swelling and protein loss in urine
• nefrotski sindrom: otekanje spodnjih okončin, otekanje po vsem telesu in izguba beljakovin z urinom
Name and address of the manufacturers of the biological active substance Genzyme Corporation 45, 51, 76, 74 and 80 New York Avenue, Framingham, MA 01701, United States
Genzyme Corporation 45, 51, 74, 76 and 80 New York Avenue, Framingham, MA 01701, Združene države Amerike
After 52 weeks of treatment, 16 of 21 patients (76.2%) treated with Myozyme were alive.
Po 52 tednih zdravljenja je bilo živih 16 izmed 21 bolnikov (76,2 %), zdravljenih z zdravilom Myozyme.
*Human acid α-glucosidase is produced in Chinese hamster ovary cells (CHO) by recombinant DNA technology.
*Humana kislina -glukozidaze se pridobiva s tehnologijo rekombinantne DNA iz celične kulture jajčnika kitajskega hrčka (CHO - Chinese Hamster Ovary).
In the above described study in 69 late-onset patients in the Netherlands, Myozyme showed an improvement in muscle strength.
V zgoraj opisani študiji na Nizozemskem je zdravilo Myozyme pri 69 bolnikih s poznim nastopom bolezni pokazalo izboljšanje moči mišic.
Myozyme contains an artificial enzyme called alglucosidase alfa - this can replace the natural enzyme which is lacking in Pompe disease.
Zdravilo Myozyme vsebuje umetni encim, ki se imenuje alglukozidaza alfa - nadomesti lahko naravni encim, ki ga primanjkuje pri Pompejevi bolezni.
Determine the number of vials to be reconstituted based on the individual patient's dose regimen (mg/kg) and remove the required vials from the refrigerator in order to allow them to reach room temperature (approximately 30 minutes).
Število vial za rekonstituiranje določite na podlagi režima odmerjanja za posameznega bolnika (mg/kg), nato potrebne viale vzemite iz hladilnika, da bodo dosegle sobno temperaturo (približno 30 minut).
However, there was no apparent association between inhibition of uptake and the co-primary efficacy endpoints (see section 4.4).
Vendar pa ni bilo očitne povezave med zaviranjem privzema in prvotnima ocenama izida učinkovitosti (glejte poglavje 4.4).
Patients presented with a constellation of signs and symptoms, primarily respiratory, cardiovascular, edematous and/or cutaneous in nature (see section 4.4).
Pri bolnikih se je pojavil skupek znakov in simptomov, ki so bili v glavnem respiratornega, kardiovaskularnega, edematoznega in/ali kožnega značaja (glejte poglavje 4.4).
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.
Pri 12 bolnikih z razpoložljivimi podatki sta bila AUC in Cmax približno enakovredna tistim, opaženim pri skupinah, ki so prejemale odmerke 20 mg/kg v osrednji raziskavi.
Younger patients diagnosed and treated early and who received ITI concomitantly to ERT initiation had a trend towards better survival rate than patients treated with similar regimen at a later age.
Mlajši bolniki, ki so bili diagnosticirani in zdravljeni zgodaj ter so prejemali ITI sočasno z uvedbo encimskega nadomestnega zdravljenja, so imeli trend k boljšemu deležu preživetja kot bolniki, zdravljeni s podobno shemo v poznejši starosti.
Adverse reaction (Preferred Term Level)
Neželeni učinek (prednostni neželeni učinek)
The reconstituted solution must be further diluted.
Rekonstituirano raztopino je treba nato razredčiti.
Other medicines and Myozyme
Druga zdravila in zdravilo Myozyme
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.
Klinično predstavitev Pompejeve bolezni je mogoče opisati kot spekter bolezni, ki sega od hitronapredujoče oblike z zgodnjim nastopom (nastop simptomov Pompejeve bolezni običajno v prvem letu življenja in zelo kratka pričakovana življenjska doba), do manj hitro-napredujoče oblike s poznim nastopom.
The probability of a poor outcome and of developing high and sustained IgG antibody titres appears higher among CRIM-negative patients (Cross Reactive Immunologic Material- negative patients in whom no endogenous GAA protein was detected by Western blot analysis) than among CRIM-positive patients in whom endogenous GAA protein was detected by Western blot analysis and/or predicted based on genotype.
Možnost nezadovoljivega izida in nastanka visokih in vzdrževanih titrov protiteles IgG je višja pri CRIM (Cross Reactive Immunologic Material) negativnih bolnikih (navzkrižno reaktivni imunološki material - negativni bolniki, pri katerih niso zaznali endogenega proteina GAA z Western blot analizo) kot pri CRIM pozitivnih bolnikih pri katerih so endogeni protein GAA ugotovili z Western blot analizo in/ali predvideli na podlagi genotipa.
However, the invasive ventilator-free survival was not recorded in the untreated historical cohort and a comparison of this endpoint is not possible.
Vendar v nezdravljeni historični kohorti preživetje brez invazivnega predihavanja ni bilo zabeleženo, tako primerjava tega cilja ni mogoča.
Patients with Pompe disease are at increased risk of respiratory infections due to the progressive effects of the disease on the respiratory muscles.
Pri bolnikih s Pompejevo boleznijo obstaja tveganje za razvoj okužbe dihal zaradi progresivnega učinka bolezni na dihalne mišice.
Side effects were mainly seen while patients were being given the medicine or shortly after (“infusion related effects”).
Neželene učinke so večinoma opazili med dajanjem tega zdravila ali takoj po njem („učinki, povezani z infundiranjem“).
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).
Po najmanj 52 tednih so 16 izmed 18 bolnikov vključili v podaljšek klinične študije, da bi nadaljevali s prejemanjem zdravila pri enakem odmerku za skupno dobo do treh let (150 tednov).
It is supplied as a powder which will be mixed with sterile water before it is given.
Na voljo je kot prašek, ki ga je treba pred dajanjem zmešati s sterilno vodo.
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.
Nato je potrebno nadaljnje redčenje, kot je opisano v nadaljevanju: Počasi odvzemajte rekonstituirano raztopino iz vsake viale, dokler ne dosežete količine za bolnikov odmerek.
Evaluation of motor function gave disparate results.
Pri ocenjevanju motoričnih funkcij so bili dobljeni različni rezultati.
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.
Po 52 tednih zdravljenja je bilo vseh 18 bolnikov, zdravljenih z zdravilom Myozyme, živih in 15 od teh ni potrebovalo podpore invazivnega predihavanja, medtem ko je bil 1 od 42 bolnikov iz nezdravljene historične kohorte živ pri 18 mesecih.
In studies doctors have used medicines to suppress the immune system to reduce the production of antibodies.
V študijah so zdravniki za zmanjšanje nastanka protiteles uporabili zdravila, ki zavirajo delovanje imunskega sistema.
People with Pompe disease have low levels of an enzyme called alpha-glucosidase.
Osebe s Pompejevo boleznijo imajo nizko raven encima, imenovanega alfa-glukozidaza.
In clinical studies, the majority of patients developed IgG antibodies to alglucosidase alfa typically within 3 months of treatment.
V kliničnih študijah je pri večini bolnikov prišlo do tvorbe protiteles IgG proti alglukozidazi alfa, običajno v treh mesecih zdravljenja.
1 Reactions reported in 39 infantile-onset patients in 2 clinical trials.
1 O učinkih so poročali pri 39 bolnikih z zgodnjim nastopom bolezni v 2 kliničnih preskušanjih.
Table 5: Alglucosidase alfa pharmacokinetics after a single dose and after 12 and 52 weeks of therapy
Tabela 5: Farmakokinetika alglukozidaze alfa po enkratnem odmerku ter po 12 in 52 tednih zdravljenja parameter
After 52 weeks all patients continued treatment for a total duration of more than 3 years (168 weeks with a median of 121 weeks).
Po 52 tednih so vsi bolniki nadaljevali z zdravljenjem s skupnim trajanjem več kot 3 leta (168 tednov z mediano 121 tednov).
Other names for Pompe disease include glycogen storage disease type II (GSDII), acid maltase deficiency (AMD) and glycogenosis type II.
Druga imena za Pompejevo bolezen so bolezen kopičenja glikogena tipa II (GSD-II), pomanjkanje kisle maltaze (AMD) in glikogenoza tipa II.
Patients treated with Myozyme demonstrated prolonged survival as compared to survival in an untreated historical cohort (see Table 2).
Pri bolnikih, zdravljenih z zdravilom Myozyme, so opazili podaljšano preživetje v primerjavi s preživetjem v nezdravljeni historični kohorti (glejte tabelo 2).
Myozyme is indicated in adults and paediatric patients of all ages.
Zdravilo Myozyme je indicirano pri odraslih in pediatričnih bolnikih vseh starosti.
Myozyme is used to treat adults, children and adolescents of all ages who have a confirmed diagnosis of Pompe disease.
Zdravilo Myozyme se uporablja za zdravljenje odraslih, otrok in mladostnikov vseh starosti s potrjeno diagnozo Pompejeve bolezni.
AUC∞ and Cmax were similar at week 0, 12 and 52 visits indicating alglucosidase alfa pharmacokinetics were not timedependent (Table 5).
Vrednosti AUC∞ in Cmax so bile podobne pri obiskih na 0., 12. in 52. teden, kar nakazuje, da farmakokinetika alglukozidaze alfa ni odvisna od časa (Tabela 5).
After 78 weeks, patients treated with Myozyme showed improvement in distance walked as measured by 6MWT and stabilization of pulmonary function as measured by FVC % predicted as compared to placebo-treated patients.
Po 78 tednih so se pri bolnikih z zdravilom Myozyme pokazale izboljšave pri prehojeni razdalji, izmerjeni s testom 6MWT, in stabilizacija delovanja pljuč, izmerjena s predvidenim % FVC, v primerjavi z bolniki, ki so prejemali placebo.
It is postulated that Myozyme will restore lysosomal GAA activity resulting in stabilisation or restoration of cardiac and skeletal muscle function (including respiratory muscles).
Domneva se, da zdravilo Myozyme ponovno vzpostavi lizosomsko aktivnost GAA, kar privede do uravnoteženja ali obnovitve delovanja srčne in skeletne mišice (vključno z dihalnimi mišicami).
After the first year (58 up to 168 weeks) of treatment LVM further decreased in 9 out of 12 patients with available data.
Po prvem letu (58 do 168 tednov) zdravljenja se je vrednost LVM še dodatno znižala pri 9 izmed 12 bolnikov, z razpoložljivimi podatki.
Date of first authorisation: 29 March 2006 Date of latest renewal: 29 March 2011
Datum zadnjega podaljšanja: 29/03/2011
Some patients have experienced infusion related side effects in the form of flu-like symptoms, which lasted for a few days after completion of the infusion.
Pri nekaterih bolnikih so se pojavili z infuzijo povezani neželeni učinki v obliki gripi podobnih simptomov, ki so bili prisotni nekaj dni po koncu infundiranja.
Your doctor should consider discontinuation of the administration of Myozyme and initiate appropriate medical treatment.
Vaš zdravnik mora presoditi o prekinitvi dajanja zdravila Myozyme in uvesti ustrezno zdravljenje.
In these patients renal biopsy showed immune complex deposition.
Pri teh bolnikih so pri biopsiji ledvic odkrili odlaganje imunskih kompleksov.
MANUFACTURERS OF THE BIOLOGICAL ACTIVE SUBSTANCE AND MANUFACTURERS RESPONSIBLE FOR BATCH RELEASE
PROIZVAJALCI BIOLOŠKE UČINKOVINE IN PROIZVAJALCI, ODGOVORNI ZA SPROŠČANJE SERIJ
Psychiatric disorders common Agitation not known
Psihiatrične motnje pogosti agitacija neznana agitacija nemir
Immunogenicity data from clinical trials and published literature in CRIM-negative infantile-onset patients (IOPD) suggests that the administration of immune tolerance induction (ITI) regimen given to alglucosidase alfa naive patients (prophylactic ITI) may be effective in preventing or reducing the development of High Sustained Antibody Titer (HSAT) against alglucosidase alfa.
Podatki o imunogenosti iz kliničnih preskušanj in objavljene literature pri CRIM-negativnih bolnikih z nastankom bolezni v obdobju dojenčka (IOPD - infantile-onset patients) kažejo, da lahko uporaba sheme za indukcijo imunske tolerance (ITI - immune tolerance induction) pri bolnikih, ki še niso prejemali alglukozidaze alfa (profilaktična ITI), učinkovito prepreči ali zmanjša pojav stalno visokega titra protiteles (HSAT - High Sustained Antibody Titer) proti alglukozidazi alfa.
Gently invert or massage the infusion bag to mix the diluted solution.
Nežno obrnite ali gnetite infuzijsko vrečko, da zmešate razredčeno raztopino.
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.
Izmed treh bolnikov s hudo pljučno prizadetostjo ob pregledu/izhodiščni vrednosti (odstotek predvidene forsirane vitalne kapacitete v sedečem položaju od 58 do 67 %) so se pri dveh do 26. tedna pokazale klinično pomembne izboljšave pri FVC (+11,5 % in +16,0 %) v sedečem položaju.
Non-clinical data reveal no special hazard for humans based on conventional studies of safety pharmacology, single and repeated dose toxicity.
Neklinični podatki na osnovi konvencionalnih študij farmakološke varnosti, toksičnosti pri enkratnih in ponavljajočih se odmerkih ne kažejo posebnega tveganja za človeka.
The vast majority of patients with infantile-onset Pompe disease treated with Myozyme demonstrate improvement in cardiac function as well as stabilisation or improvements in growth parameters.
Pri veliki večini bolnikov, z zgodnjim nastopom Pompejeve bolezni, zdravljenih z zdravilom Myozyme, je bilo dokazano izboljšanje srčne funkcije, kot tudi uravnoteženje ali izboljšanje parametrov rasti.
Myozyme will be given to you under the supervision of a doctor who is experienced in the treatment of Pompe disease.
Zdravilo Myozyme boste dobili pod nadzorom zdravnika, ki ima izkušnje z zdravljenjem Pompejeve bolezni.
Due to the proteinaceous nature of the product, particle formation may occur in the reconstituted solution and final infusion bags.
Zaradi beljakovinske narave zdravila lahko pride do tvorbe delcev v rekonstituirani raztopini in končnih infuzijskih vrečkah.
The primary endpoint was the proportion of patients who were alive and free of invasive ventilator support.
Glavni cilj je bil delež živih bolnikov, ki niso potrebovali podpore invazivnega predihavanja.
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.
V študiji retrospektivne analize podatkov pri bolnikih z zgodnjim nastopom Pompejeve bolezni (n=168) je bila srednja starost ob nastopu simptomov 2,0 meseca in srednja starost ob smrti 9,0 mesecev.
Comparison of survival curves from time of diagnosis versus the untreated historical cohort was made using a Cox proportional hazards regression analysis.
Primerjava krivulje preživetja od časa diagnoze s krivuljo nezdravljene historične kohorte je bila opravljena s Coxovo regresijsko analizo sorazmernih tveganj.
What Myozyme is and what it is used for What you need to know before you are given Myozyme How Myozyme is given
Kaj je zdravilo Myozyme in za kaj ga uporabljamo Kaj morate vedeti, preden boste prejeli zdravilo Myozyme Kako uporabljati zdravilo Myozyme Možni neželeni učinki
Slightly more patients in the Myozyme group than in the placebo group experienced IARs (28% versus 23%).
IARs so se pojavile pri malce več bolnikih v skupini z zdravilom Myozyme kot v skupini s placebom (28 % napram 23 %).
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.
Pompejeva bolezen s poznim nastopom se pojavlja pri dojenčkih, otrocih, mladostnikih ali celo odraslih in napreduje precej počasneje kot oblika z zgodnjim nastopom.
Fatal and life-threatening respiratory infections have been observed in some of these patients.
Pri nekaterih od teh bolnikov so opazili smrtne in življenjsko nevarne okužbe dihal.
Life threatening hypersensitivity (anaphylactic reaction) to the active substance or to any of the excipients listed in section 6.1, when rechallenge was unsuccessful (see sections 4.4 and 4.8).
Življenjsko nevarna preobčutljivost (anafilaktična reakcija) na učinkovino ali katerokoli pomožno snov, navedeno v poglavju 6.1, kjer je bilo ponovno zdravljenje neuspešno (glejte poglavji 4.4 in 4.8).
Add the water for injections by slow drop-wise addition down the side of the vial and not directly onto the lyophilised cake.
Vodo za injekcije dodajajte s počasnim spuščanjem kapljic po steni viale in ne neposredno na liofilizirano pogačo.
Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency).
Zdravilo Myozyme je namenjeno dolgoročni encimski nadomestni terapiji (ERT - enzyme replacement therapy) pri bolnikih s potrjeno diagnozo Pompejeve bolezni (pomanjkanje glukozidaze).
The totality of the data suggests that early diagnosis and treatment at an early stage of disease may be critical to achieve the best outcomes in these infantile onset patients.
Celota podatkov pri teh bolnikih z zgodnjim nastopom bolezni kaže, da sta za dosego najboljših rezultatov pomembna zgodnja diagnoza in zdravljenje v zgodnjem stadiju bolezni.
Adverse reactions were mostly mild to moderate in intensity and almost all occurred during the infusion or during the 2 hours following the infusion (infusion associated reactions, IARs).
Neželeni učinki so bili najpogosteje blagi do zmerni in skoraj vsi so se pojavili v času infuzije ali v dveh urah po prejemu infuzije (IAR - z infuzijo povezane reakcije).
There is no experience of the use of Myozyme in pregnant women.
Ni izkušenj z uporabo zdravila Myozyme pri nosečnicah.
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.
Pompejeva bolezen spada med motnje lizosomskega shranjevanja, ki jih povzroči pomanjkanje naravne lizosomske hidrolaze, kisle α-glukozidaze (GAA), ki razgradi lizosomski glikogen do glukoze.
One vial contains 50 mg of alglucosidase alfa.
Ena viala vsebuje 50 mg alglukozidaze alfa.
The % predicted FVC changed by a median of 0.0 for Myozyme-treated patients and decreased by a median of 3% for placebo-treated patients, indicating a statistically significant treatment effect (p=0.0026).
Predvideni % FVC se je spremenil za povprečno 0,0 pri bolnikih, zdravljenih z zdravilom Myozyme, in znižal za povprečno 3 % pri bolnikih, zdravljenih s placebom, kar nakazuje na statistično pomemben učinek zdravljenja (p=0,0026).
Patients with an acute illness (e.g. pneumonia, sepsis) at the time of Myozyme infusion appear to be at greater risk for IARs.
Bolniki z akutno boleznijo (npr. pljučnico, sepso) so v času infuzije zdravila Myozyme izpostavljeni večjemu tveganju za infuzijske reakcije.
Patients were randomized in a 2:1 ratio and received 20 mg/kg Myozyme (n=60) or placebo (n=30) once every two weeks for 78 weeks (18 months).
Bolniki so bili naključno razporejeni v razmerju 2:1 ter so prejemali 20 mg/kg zdravila Myozyme (n=60) ali placebo (n=30) enkrat na vsaka dva tedna v obdobju 78 tednov (18 mesecev).
Subjects were aged 6 months or less at the onset of treatment.
Ob pričetku zdravljenja so bile osebe stare 6 mesecev ali manj.
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.
Retrospektiven pregled dokumentacije v centru Duke je odkril 21 CRIM-negativnih bolnikov z IOPD; od teh jih 19 do uvedbe ITI še nikdar ni prejemalo encimskega nadomestnega zdravljenja.
Approximately half of the patients treated with Myozyme in infantile-onset clinical studies and 28% of the patients treated with Myozyme in a late-onset clinical study developed infusion associated reactions (IARs).
Pri približno polovici bolnikov, zdravljenih z zdravilom Myozyme v kliničnih študijah zgodnjega nastopa bolezni in 28 % bolnikov, zdravljenih z zdravilom Myozyme v kliničnih študijah poznega nastopa bolezni, so se pojavile infuzijske reakcije (IARs).
IARs are defined as any related adverse event occurring during the infusion or during the hours following infusion.
IARs so opredeljene kot katerikoli neželeni učinek, povezan z zdravilom, ki nastopi med infundiranjem ali nekaj ur po infuziji.
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.
Boljše odzive na zdravljenje so opažali pri mlajših bolnikih z manj napredovalo boleznijo, ki so prejemali profilaktično ITI pred pojavom HSAT; to kaže, da lahko zgodnja uvedba ITI izboljša klinične izide.
You are recommended to stop breast-feeding when you are given Myozyme.
Priporočamo vam, da pri dajanju zdravila Myozyme prenehate z dojenjem.
Table 2: Results for endpoint survival using the Cox regression model
Tabela 2: Rezultati preživetja po Coxovem regresijskem modelu
A limited number of the IgG positive patients evaluated tested positive for inhibitory effects on in vitro testing.
Pri majhnem številu IgG pozitivnih bolnikov, ki so bili ocenjevani, je bil test za zaviralne učinke pri in vitro preizkusih pozitiven.
At the end of the study (with individual patient treatment durations ranging from 60 to 150 weeks; mean follow-up period of 119 weeks) 14 of 16 patients were alive and 9 of 16 patients were alive and free of invasive ventilatory support.
Ob zaključku študije (s trajanjem zdravljenja pri posameznih bolnikih v razponu med 60 in 150 tednov; povprečno obdobje sledenja je bilo 119 tednov) je bilo 14 izmed 16 bolnikov živih ter 9 izmed 16 bolnikov je bilo živih in ni potrebovalo podpore invazivnega predihavanja.
Infusion Associated Reactions
Infuzijske reakcije
Table 3: Results for endpoint survival using the Cox regression model
Tabela 3: Rezultati preživetja po Coxovem regresijskem modelu
N=21 N=48
n=48
The risks and benefits of re-administering alglucosidase alfa following an immune-mediated reaction should be considered.
Presoditi morate tveganja in koristi ponovne uporabe alglukozidaze alfa po imunsko posredovani reakciji.
Serious adverse reactions reported in 4 patients treated with Myozyme were: angioedema, chest discomfort, throat tightness, non-cardiac chest pain and supraventricular tachycardia.
Resni neželeni učinki, o katerih so poročali pri 4 bolnikih, zdravljenih z zdravilom Myozyme, so bili: angioedem, neugodje v prsih, stiskanje v grlu, bolečina v prsih (ne v srcu) in superventrikularna tahikardija.
The untreated historical cohort was matched to the pivotal study population and was derived from a retrospective natural history study (n=42) in patients with infantile-onset Pompe disease.
Nezdravljena historična kohorta se je ujemala s populacijo osrednje študije in bila pridobljena iz retrospektivne analize podatkov (n=42) bolnikov z zgodnjim nastopom Pompejeve bolezni.
You should not be given Myozyme during pregnancy unless clearly necessary.
Med nosečnostjo zdravila Myozyme ne smete dobiti, razen če je to nujno potrebno.