natgillin/translations-raw huggingface dataset:
It is possible that your child has no side effects from vaccination.
Možno je, da otrok po cepljenju ne bo imel nobenih neželenih učinkov.
If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor or pharmacist.
Če kateri koli neželeni učinek postane resen, ali če opazite kateri koli neželeni učinek, ki ni omenjen v tem navodilu, obvestite svojega zdravnika ali farmacevta.
HOW TO STORE AMBIRIX
SHRANJEVANJE CEPIVA AMBIRIX
Keep out of the reach and sight of children.
Cepivo shranjujte nedosegljivo otrokom!
Do not use Ambirix after the expiry date which is stated on the carton.
Cepiva Ambirix ne smete uporabljati po datumu izteka roka uporabnosti, ki je naveden na škatli.
What Ambirix contains
Kaj vsebuje cepivo Ambirix
- The active substances are:
- Zdravilni učinkovini sta:
Hepatitis A virus (inactivated)1,2
Virus hepatitisa A (inaktivirani) 1, 2 Površinski antigen virusa hepatitisa B3, 4 1 Pridobljen na humanih diploidnih celicah (MRC- 5) 2
720 ELISA Units
720 ELISA enot 20 mikrogramov
Produced in yeast cells (Saccharomyces cerevsiae) by recombinant DNA technology 4
0, 05 miligrama Al3+ Adsorbiran na hidratirani aluminijev hidroksid 3 Pridobljen s tehnologijo rekombinantne DNA na celični kulturi kvasovk (Saccharomyces cerevisiae) 4 0, 4 miligrama Al3+ Adsorbiran na aluminijev fosfat
- The other ingredients in Ambirix are: sodium chloride and water for injections.
- Pomožne snovi cepiva Ambirix so: natrijev klorid in voda za injekcije.
(1 ml)
Izgled cepiva Ambirix in vsebina pakiranja
Ambirix is available in packs of 1 and 10 with or without needles and in a pack size of 50 without needles.
Suspenzija za injiciranje (1 ml) Cepivo Ambirix je na voljo v pakiranjih po 1 in 10 z injekcijskimi iglami ali brez njih ter po 50 brez injekcijskih igel.
Ambirix is a white, slightly milky liquid presented in a prefilled syringe.
Cepivo Ambirix je bela, rahlo mlečna tekočina v napolnjeni injekcijski brizgi.
Rue de l’Institut 89 B-1330 Rixensart Belgium
Rue de l’ Institut 89 B- 1330 Rixensart Belgija
Deutschland GlaxoSmithKline GmbH & Co.
23 Deutschland GlaxoSmithKline GmbH & Co.
Detailed information on this medicine is available on the European Medicines Agency (EMEA) web site: http://www.emea.europa.eu/.
24 Podrobne informacije o zdravilu so objavljene na spletni strani Evropske agencije za zdravila (EMEA) http: // www. emea. europa. eu /.
25 Upon storage, a fine white deposit with a clear colourless supernatant can be observed.
Med shranjevanjem se lahko pojavi fina bela usedlina in bister brezbarven supernatant.
The vaccine should be visually inspected before or after resuspension for any foreign particulate matter and/or change in physical appearance The vaccine must not be used if any change in the appearance of the vaccine has taken place.
Če se opazi kakršna koli sprememba v videzu, se cepivo ne sme uporabiti.
Any unused vaccine or waste material should be disposed of in accordance with local requirements.
Vsako neuporabljeno cepivo ali odpadni material je treba zavreči v skladu z lokalnimi predpisi.
26 ANNEX IV
26 PODLAGA ZA ENO DODATNO PODALJŠANJE
GROUNDS FOR ONE ADDITIONAL RENEWAL
Redna poročila o varnosti zdravila (PSURs) se morajo še dalje zagotavljati na letni osnovi.
It explains how the Committee for Medicinal products for Human Use (CHMP) assessed the studies performed, to reach their recommendations on how to use the medicine.
Ta dokument je povzetek Evropskega javnega poročila o oceni zdravila (EPAR). Pojasnjuje, kako je Odbor za zdravila za uporabo v humani medicini (CHMP) ocenil opravljene raziskave, na podlagi česar je oblikoval priporočila glede uporabe zdravila.
Ammonaps contains the active substance sodium phenylbutyrate.
Zdravilo Ammonaps vsebuje zdravilno učinkovino natrijev fenilbutirat.
It is available as white oval tablets or as granules, given orally.
Na voljo je v obliki belih ovalnih tablet ali zrnc za peroralno uporabo.
Ammonaps is used to reduce levels of ammonia and glutamine in the blood.
Zdravilo Ammonaps se uporablja za znižanje ravni amoniaka in glutamina v krvi.
Ammonaps is used with other treatments and a special diet for the long-term management of patients with urea cycle disorders where there is lack of one or more of the following enzymes: carbamyl phosphate synthetase, ornithine transcarbamylase, or argininosuccinate synthetase.
Zdravilo Ammonaps se uporablja skupaj z drugimi zdravili in posebno prehrano za dolgoročno zdravljenje bolnikov z motnjami ciklusa sečnine s pomanjkanjem enega ali več izmed naslednjih encimov: karbamil- fosfat- sintetaze, ornitin- transkarbamilaze ali argininosukcinat- sintetaze.
Patients with these rare disorders lack certain liver enzymes and are therefore not able to get rid of nitrogen waste, which builds up after eating protein.
Bolniki s temi redkimi motnjami nimajo določenih jetrnih encimov in se zato ne morejo znebiti odpadnega dušika, ki se začne kopičiti po zaužitju beljakovin.
Nitrogen waste is in the form of ammonia, which is especially toxic for the brain.
Odpadni dušik je v obliki amoniaka, ki je še zlasti strupen za možgane.
Ammonaps is used for babies with a complete lack of enzymes within 28 days of being born.
Zdravilo Ammonaps uporabljamo pri dojenčkih s popolnim pomanjkanjem encimov v roku 28 dni po rojstvu.
Ammonaps can also be used in patients over one month old who have partial lack of enzymes or have a history of hyperammonaemic encephalopathy (brain damage caused by high levels of ammonia).
Zdravilo Ammonaps lahko uporabljamo tudi pri bolnikih, starejših od enega meseca, ki imajo delno pomanjkanje encimov ali so preboleli hiperamonemično encefalopatijo (poškodba možganov, ki jo povzroči previsoka raven amoniaka).
A doctor who has experience in treating patients with urea cycle disorders should start the treatment with Ammonaps.
Zdravljenje z zdravilom Ammonaps naj uvede zdravnik, izkušen v zdravljenju bolnikov z motnjami ciklusa sečnine.
The daily dose depends on the diet, body weight or body surface, and amount of protein in the diet.
Dnevni odmerek je odvisen od prehrane, telesne mase ali telesne površine ter količine beljakovin v prehrani.
Ammonaps is taken in divided doses at meal times, by mouth, mixed with food or through a gastrostomy (tube through the abdomen to the stomach) or nasogastric tube (tube through the nose to the stomach).
Zdravilo Ammonaps se jemlje v enakomerno razdeljenih odmerkih ob obrokih, peroralno, pomešano s hrano ali prek gastrostome (cevke, ki vodi skozi trebušno steno v želodec) ali nazogastrične sonde (cevke, ki vodi skozi nos v želodec).
The active substance in Ammonaps, sodium phenylbutyrate, is transformed by the body into another compound, phenylacetate.
Zdravilno učinkovino v zdravilu Ammonaps, natrijev fenilbutirat, telo pretvori v drugo spojino, fenilacetat.
Phenylacetate combines with glutamine, and is eliminated by the kidneys from the body, taking with it the nitrogen waste.
Fenilacetat se veže z glutaminom, iz telesa se izloči prek ledvic in s seboj odnese tudi odpadni dušik.
(44-20) 74 18 84 00 Fax (44-20) 74 18 86 68 E-mail: mail@emea. europa. eu http: / /www. emea. europa. eu ©EMEA 2006 Reproduction and/ or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged to a placebo (dummy treatment).
(44- 20) 74 18 84 00 Fax (44- 20) 74 18 86 68 E- mail: mail@ emea. europa. eu http: // www. emea. europa. eu ©EMEA 2006 Reproduction and/ or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged Kako je bilo zdravilo Ammonaps raziskano?
The main measurement was survival.
Glavno merilo je bilo preživetje.
Other things studied were the number of hyperammonaemic episodes, cognitive (intelligence) development, growth, and blood ammonia and glutamine levels.
Preučevali so tudi število hiperamoniemičnih epizod, kognitivni (inteligenca) razvoj, rast ter ravni amoniaka in glutamina v krvi.
What benefit has Ammonaps shown during the studies?
Kakšne koristi je zdravilo Ammonaps izkazalo med raziskavami?
The overall survival rate was about 80% for the disease in newborn babies.
Povprečna stopnja preživetja je bila približno 80% za bolezen pri novorojenčkih.
The survival rate was higher in patients who had developed the disease later in life.
Stopnja preživetja je bila višja pri bolnikih, ki so bolezen razvili kasneje v življenju.
About a quarter of female patients will be affected by the most common side effects.
Najpogostejši neželeni učinki prizadenejo približno četrtino bolnic.
These are irregular menstrual periods or stopped menstrual periods (although stopped periods may be due to pregnancy).
Ti so neredne menstruacije ali izostanek le- teh (izostanek menstruacije je lahko tudi posledica nosečnosti).
Other common side effects include abnormal kidney function and blood cell counts (red cells, white cells, platelets).
Drugi pogosti neželeni učinki vključujejo nenormalno delovanje ledvic in nenormalne koncentracije krvnih celic (rdečih krvničk, belih krvničk, krvnih ploščic).
Ammonaps may also cause decreased appetite, abnormal blood acidity, depression, irritability, headache, fainting, fluid retention, bad taste or taste aversion, abdominal pain, vomiting, nausea, constipation, rash, unpleasant body odour, or weight gain.
Zdravilo Ammonaps lahko povzroči tudi zmanjšanje teka, nenormalno kislost krvi, depresijo, razdražljivost, glavobol, omedlevico, zastoj tekočin, slab okus ali odpor do določenega okusa, bolečine v trebuhu, bruhanje, slabost, zaprtje, izpuščaje, neprijetni telesni vonj ali povečanje telesne teže.
Ammonaps should not be used in people who may be hypersensitive (allergic) to sodium phenylbutyrate or any of the other ingredients.
Zdravila Ammonaps ne smejo uporabljati osebe, ki utegnejo biti preobčutljive za (alergične na) natrijev fenilbutirat ali katerokoli drugo sestavino.
Female patients who received Ammonaps must ensure that they do not become pregnant.
Bolnice, ki so prejele zdravilo Ammonaps, morajo poskrbeti, da ne zanosijo.
The Committee for Medicinal products for Human Use (CHMP) decided that Ammonaps effectively prevents ammonia levels to become too high in patients who have urea cycle disorders.
Odbor za zdravila za uporabo v humani medicini (CHMP) je odločil, da zdravilo Ammonaps učinkovito preprečuje prevelik porast vrednosti amoniaka v krvi pri bolnikih z motnjami ciklusa sečnine.
This is a serious disease and there are very few treatments.
To je resna bolezen, možnosti zdravljenja pa zelo malo.
The CHMP decided that Ammonaps’ benefits are greater than its risks and recommended that Ammonaps be given marketing authorisation.
Odbor za zdravila za uporabo v humani medicini (CHMP) je odločil, da so koristi zdravila Ammonaps večje od z njim povezanih tveganj, in priporočil, da se za zdravilo Ammonaps odobri dovoljenje za promet.
Ammonaps was authorised under "Exceptional Circumstances", because, as the disease is rare, limited information was available at the time of approval.
Dovoljenje za promet z zdravilom za zdravilo Ammonaps je bilo odobreno v “ izjemnih okoliščinah ”, ker so bili ob času odobritve na voljo le omejeni podatki, saj je bolezen zelo redka.
As the company had supplied the additional information requested, the "Exceptional Circumstances" ended on6 July 2004.
Podjetje je predložilo zahtevane dodatne informacije, zato je od 6. julija 2004 omejitev » v izjemnih okoliščinah « preklicana.
The marketing authorisation holder is Swedish Orphan International AB.
Dovoljenje za promet z zdravilom je bilo podaljšano dne 8. decembra 2004. imetnik dovoljenja za promet z zdravilom je Swedish Orphan International AB.
The full EPAR for Ammonaps is available here.
Celotno Evropsko javno poročilo o oceni zdravila (EPAR) za zdravilo Ammonaps je na voljo tukaj.
This summary was last updated in 10-2006.
Ta povzetek je bil nazadnje posodobljen 10- 2006.
The tablets are off-white, oval and embossed with “ UCY 500”.
Tablete so sivo bele, ovalne in imajo vrezan napis “ UCY 500 ”.
AMMONAPS is indicated as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.
Zdravilo AMMONAPS uporabljamo kot dodatno zdravljenje pri dolgoročnem obravnavanju motenj ciklusa sečnine, ki vključujejo pomanjkljivosti karbamoilfosfat- sintaze, ornitin transkarbamoilaze ali argininosukcinat- sintaze.
It is indicated in all patients with neonatal-onset presentation (complete enzyme deficiencies, presenting within the first 28 days of life).
Zdravilo AMMONAPS je indicirano pri vseh bolnikih, pri katerih se bolezen pojavi s t. i. neonatalnim pojavom (celotne pomanjkljivosti encimov, ki se pokažejo v prvih 28 dneh življenja).
It is also indicated in patients with late-onset disease (partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy.
Indicirano je tudi pri bolnikih, pri katerih se bolezen pojavi s t. i. zakasnjenim pojavom (delne pomanjkljivosti encimov, ki se pokažejo po prvem mesecu življenja) in imajo anamnezo hiperamonemične encefalopatije.
The use of AMMONAPS tablets is indicated for adults and children who are able to swallow tablets.
Jemanje tablet AMMONAPS je indicirano pri odraslih in otrocih, ki lahko požirajo tablete.
The daily dose should be individually adjusted according to the patient ’ s protein tolerance and the daily dietary protein intake needed to promote growth and development.
Dnevni odmerek se mora prilagoditi individualno glede na bolnikovo toleranco za beljakovine in na potreben dnevni vnos beljakovin za pospeševanje rasti in razvoja.
The safety and efficacy of doses in excess of 20 g/ day (40 tablets) have not been established.
Običajni celokupni dnevni odmerek natrijevega fenilbutirata je po kliničnih izkušnjah: • 450 – 600 mg/ kg/ dan pri otrocih, ki tehtajo manj kot 20 kg 9, 9 – 13, 0 g/ m2/ dan pri otrocih, ki tehtajo več kot 20 kg, ter pri mladostnikih in pri odraslih. • Varnost in učinkovitost odmerkov večjih od 20 g/ dan (40 tablet), še ni bila dokazana.
The total daily dose should be divided into equal amounts and given with each meal (e. g. three times per day).
Celokupni dnevni odmerek naj se razdeli na enake dele in se daje z vsakim obrokom (npr. trikrat dnevno).
AMMONAPS tablets should be taken with a large volume of water.
Tablete AMMONAPS je treba jemati z veliko količino vode.
Therapeutic monitoring:
Nadzorovanje zdravljenja:
Plasma levels of ammonia, arginine, essential amino acids (especially branched chain amino acids), carnitine and serum proteins should be maintained within normal limits.
Plazemske koncentracije amoniaka, arginina, esencialnih aminokislin (predvsem razvejane verige aminokislin), karnitina in serumskih beljakovin se morajo vzdrževati v normalnih mejah.
Plasma glutamine should be maintained at levels less than 1,000 µmol/ l.
Plazemske koncentracije glutamina se morajo vzdrževati pod 1. 000 µmol/ l.
Nutritional management:
2 Prehrana bolnika:
AMMONAPS must be combined with dietary protein restriction and, in some cases, essential amino acid and carnitine supplementation.
Zdravilo AMMONAPS moramo kombinirati z omejitvami prehranskega vnosa beljakovin in v določenih primerih tudi s prehranskimi dopolnili esencialnih aminokislin in karnitina.
2 Citrulline or arginine supplementation is required for patients diagnosed with neonatal-onset form of carbamyl phosphate synthetase or ornithine transcarbamylase deficiency at a dose of 0.17 g/ kg/ day or 3.8 g/ m2/ day.
Za bolnike z diagnozo neonatalnega pojava pomanjkljivosti karbamoilfosfat- sintaze je potrebno dopolnjevanje prehrane s citrulinom v odmerku 0, 17 g/ kg/ dan oziroma pri pomanjkljivosti ornitin transkarbamoilaze pa je potrebno dopolnjevanje prehrane z argininom v odmerku 3, 8 g/ m2/ dan.
Arginine supplementation is required for patients diagnosed with deficiency of argininosuccinate synthetase at a dose of 0.4-0.7 g/ kg/ day or 8.8 - 15.4 g/ m2/ day.
Dopolnjevanje prehrane z argininom je potrebno za bolnike z diagnozo pomanjkljivosti argininosukcinat- sintaze, in sicer z odmerki 0, 4 – 0, 7 g/ kg/ dan ali 8, 8 – 15, 4 g/ m2/ dan.
− Pregnancy − Breast-feeding − Hypersensitivity to the active substance or to any of the excipients.
Preobčutljivost za zdravilno učinkovino ali katerokoli pomožno snov. −
AMMONAPS tablets should not be used in patients with dysphagia due to the potential risk of oesophageal ulceration if tablets are not promptly delivered to the stomach.
Zaradi možne nevarnosti, da bi nastala ulceracija požiralnika, če tablete ne bi takoj prispele v želodec, tablet AMMONAPS pri bolnikih z disfagijo ni dovoljeno uporabljati.
Each AMMONAPS tablet contains 62 mg (2.7 mmol) of sodium, corresponding to 2.5 g (108 mmol) of sodium per 20 g of sodium phenylbutyrate, which is the maximum daily dose.
Vsaka tableta zdravila AMMONAPS vsebuje 62 mg (2, 7 mmol) natrija, kar ustreza 2, 5 g (108 mmol) natrija v maksimalnem dnevnem odmerku natrijevega fenilbutirata, ki znaša 20 g.
Since the metabolism and excretion of sodium phenylbutyrate involves the liver and kidneys, AMMONAPS should be used with caution in patients with hepatic or renal insufficiency.
Ker presnova in izločanje natrijevega fenilbutirata vključuje jetra in ledvica, je potrebna previdnost pri predpisovanju AMMONAPSA bolnikom z motnjami v delovanju jeter in ledvic.
Concurrent administration of probenicid may affect renal excretion of the conjugation product of sodium phenylbutyrate.
Sočasno dajanje probenecida lahko vpliva na izločanje konjugiranega produkta natrijevega fenilbutirata preko ledvic.
Evaluation of experimental animal studies has shown reproductive toxicity, i. e. effects on the development of the embryo or the foetus.
Študije na živalih so pokazale reproduktivno toksičnost, tj. učinkovanje na razvoj zarodka ali ploda.
Prenatal exposure of rat pups to phenylacetate (the active metabolite of phenylbutyrate) produced lesions in cortical pyramidal cells; dendritic spines were longer and thinner than normal and reduced in number.
Pomen tega
The significance of these data in pregnant women is not known; therefore the use of AMMONAPS is contra-indicated during pregnancy (see section 4.3).
Ženske v rodni dobi morajo poskrbeti za učinkovito kontracepcijsko zaščito.
When high doses of phenylacetate (190 - 474 mg/ kg) were given subcutaneously to rat pups, decreased proliferation and increased loss of neurons were observed, as well as a reduction in CNS myelin.
Skrčil se je tudi mielin v CŽS.
It has not been determined if phenylacetate is secreted in human milk and therefore the use of AMMONAPS is contra-indicated during lactation (see section 4.3).
Ni znano ali fenilacetat prehaja v materino mleko, zato je uporaba zdravila AMMONAPS med dojenjem kontraindicirana (glejte poglavje 4. 3).
In clinical trials with AMMONAPS, 56% of the patients experienced at least one adverse event and 78% of these adverse events were considered as not related to AMMONAPS.
V kliničnih preskušanjih z zdravilom AMMONAPS je 56% bolnikov doživelo najmanj en neželeni dogodek, vendar se za 78% teh neželenih dogodkov smatra, da niso povezani z zdravilom AMMONAPS.
Frequency is defined as very common (≥ 1/ 10), common (≥ 1/ 100 to < 1/ 10) and uncommon (≥ 1/ 1,000 to < 1/ 100).
Pogostnost pojavljanja je definirana z zelo pogosto (≥1/ 10), pogosto (≥1/ 100 do < 1/ 10) in občasno (≥1/ 1. 000 do < 1/ 100).
Decreased blood potassium, albumin, total protein and phosphate.
Preiskave Pogosto: zmanjšana koncentracija kalija, albumina, celokupnih beljakovin in fosfata v krvi.
Arrhythmia
Srčne bolezni Pogosto: edem Občasno: aritmija
Aplastic anaemia, ecchymosis
Bolezni krvi in limfatičnega sistema Pogosto: anemija, trombocitopenija, levkopenija, levkocitoza, trombocitoza Občasno: aplastična anemija, ekhimoza
Syncope, headache
Bolezni živčevja Pogosto: sinkopa, glavobol
Pancreatitis, peptic ulcer, rectal haemorrhage, gastritis
Bolezni prebavil Pogosto: bolečine v trebuhu, bruhanje, navzea, zaprtost, motnje okušanja Občasno: pankreatitis, želodčni ulkus, rektalna krvavitev, gastritis
Renal tubular acidosis
Bolezni sečil Pogosto: acidoza ledvičnih tubulov
Rash, abnormal skin odour
Bolezni kože in podkožja Pogosto: izpuščaj, nenavaden telesni vonj
Metabolic acidosis, alkalosis, decreased appetite
Presnovne in prehranske motnje Pogosto: metabolična acidoza, alkaloza, zmanjšan apetit
Amenorrhoea, irregular menstruation
Motnje reprodukcije in dojk Zelo pogosto: izostanek menstruacije, neredna menstruacija
Depression, irritability
4 Psihiatrične motnje Pogosto: depresija, preobčutljivost
4 A probable case of toxic reaction to AMMONAPS (450 mg/ kg/ d) was reported in an 18-year old anorectic female patient who developed a metabolic encephalopathy associated with lactic acidosis, severe hypokalaemia, pancytopaenia, peripheral neuropathy, and pancreatitis.
Poročali so o verjetnem primeru toksične reakcije na zdravilo AMMONAPS (450 mg/ kg/ d) pri 18- letni anorektični bolnici, pri kateri se je razvila metabolična encefalopatija, povezana z laktično acidozo, težko hipokaliemijo, pancitopenijo, periferno nevropatijo in pankreatitisom.
One case of overdose occurred in a 5-month old infant with an accidental single dose of 10 g (1370 mg/ kg).
Do prevelikega odmerjanja je prišlo pri petmesečnem dojenčku z naključnim zaužitjem enkratnega odmerka 10 g (1370 mg/ kg).
These symptoms are consistent with the accumulation of phenylacetate, which showed dose-limiting neurotoxicity when administered intravenously at doses up to 400 mg/ kg/ day.
Ti simptomi so skladni z nakopičenjem fenilacetata, za katerega je bila dokazana nevtrotoksičnost odvisna od intravenskega odmerka pri odmerkih 400 mg/ kg/ dan.
Pharmacotherapeutic group: alimentary tract and metabolism product, ATC code:
Zdravila za bolezni prebavil in presnove, oznaka ATC:
Phenylacetate is a metabolically active compound that conjugates with glutamine via acetylation to form phenylacetylglutamine which is then excreted by the kidneys.
Le- tega nato izločajo ledvice.
Based on studies of phenylacetylglutamine excretion in patients with urea cycle disorders it is possible to estimate that, for each gram of sodium phenylbutyrate administered, between 0.12 and 0.15 g of phenylacetylglutamine nitrogen are produced.
Na podlagi študij izločanja fenilacetilglutamina pri bolnikih z motnjami ciklusa sečnine, lahko ocenimo, da se na vsak odmerjen gram natrijevega fenilbutirata proizvede med 0, 12 in 0, 15 g dušika v obliki fenilacetilglutamina.
With haemodialysis, use of alternative waste nitrogen excretion pathways (sodium phenylbutyrate, sodium benzoate and sodium phenylacetate), dietary protein restriction, and, in some cases, essential amino acid supplementation, the survival rate in new-borns diagnosed after birth (but within the first month of life) increased to almost 80% with most deaths occurring during an episode of acute hyperammonaemic encephalopathy.
S hemodializo, uporabo alternativnih poti izločanja odpadnega dušika (natrijev fenilbutirat, natrijev benzoat in natrijev fenilacetat), omejevanjem prehranskega vnosa beljakovin in (v določenih primerih) dopolnili esencialnih aminokislin se je stopnja preživetja pri novorojenčkih s postavitvijo diagnoze v prvem mesecu življenja povečala na skoraj 80%; pri čemer do večine smrti pride med epizodo hiperamoniemične encefalopatije.
In patients diagnosed during gestation and treated prior to any episode of hyperammonaemic encephalopathy, survival was 100%, but even in these patients, many subsequently demonstrated cognitive impairment or other neurologic deficits.
Toda tudi pri velikem deležu teh bolnikov so kasneje odkrili kognitivne motnje ali druge nevrološke pomanjkljivosti.