natgillin/translations-raw huggingface dataset:
The largest study of Orfadin has been in 257 patients in 87 different hospitals in 25 countries, as part of a ‘ compassionate use’ programme (this is a programme through which doctors can request a medicine for one of their patients before the medicine is fully authorised).
Največja raziskava zdravila Orfadin je potekala na 257 bolnikih v 87 različnih bolnicah v 25 državah kot del programa “ sočutne uporabe ” (to je program, v okviru katerega lahko zdravniki zahtevajo zdravilo za enega izmed svojih bolnikov, preden je zdravilo v celoti odobreno).
The effect of Orfadin on 7 Westferry Circus, Canary Wharf, London E14 4HB, UK Tel.
Učinek zdravila 7 Westferry Circus, Canary Wharf, London E14 4HB, UK Tel.
(44-20) 74 18 84 00 Fax (44-20) 74 18 84 16 E-mail: mail@emea. eu. int http: / /www. emea. eu. int ©EMEA 2006 Reproduction and/ or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged survival was studied, and compared to historical records (report of patient survival with diet only as published in medical journals).
(44- 20) 74 18 84 00 Fax (44- 20) 74 18 84 16 E- mail: mail@ emea. eu. int http: // www. emea. eu. int ©EMEA 2006 Reproduction and/ or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged Orfadin na preživetje so preučevali in primerjali s predhodnimi evidencami (poročili o preživetju bolnikov z dieto, objavljenimi v medicinskih revijah).
What benefit has Orfadin shown during the studies?
Kakšne koristi je zdravilo Orfadin izkazalo med raziskavami?
The main benefit is that Orfadin has been shown to greatly extend life.
Glavna korist, ki jo je izkazalo zdravilo Orfadin, je to, da močno podaljša življenje.
For example, a baby less than 2 months old with HT-1 would normally have only a 28% chance of surviving 5 years using diet alone.
Na primer, dojenček s HT- 1, star manj kot 2 meseca, ima ponavadi le 28- odstotno verjetnost preživetja v trajanju 5 let, če uporablja samo dieto.
With additional Orfadin treatment, the survival rate increases to 82%.
Z dodatno terapijo z zdravilom Orfadin, stopnja preživetja naraste na 82%.
The sooner treatment is started, the better the rate of survival.
Prej ko začnemo z zdravljenjem, višja je stopnja preživetja.
The most common side effects of Orfadin are the result of high tyrosine levels caused by patients not eating the right foods as well as rare cases of a decrease in platelet and white blood cell counts.
Najpogostejši neželeni učinki zdravila Orfadin so posledica visokih koncentracij tirozina, do katerih pride zaradi tega, ker bolniki ne uživajo ustrezne hrane, in v redkih primerih tudi znižanje števila trombocitov in belih krvnih celic.
For the full description of the side effects reported with Orfadin, please see the package leaflet.
Celovit opis neželenih učinkov, o katerih so poročali pri uporabi zdravila Orfadin, najdete v Navodilih za uporabo.
Orfadin should not be used in people who may be hypersensitive (allergic) to nitisinone or any of the other ingredients.
Zdravila Orfadin ne smejo uporabljati osebe, ki utegnejo biti preobčutljive za (alergične na) nitizinon ali katerokoli drugo sestavino.
The Committee for Medicinal products for Human Use (CHMP) decided that Orfadin seems to be an effective treatment of HT-1, particularly if started early before the patient ’ s liver is too damaged, and it provides a better outcome for patients than that reported in literature with diet alone.
Odbor za zdravila za uporabo v humani medicini (CHMP) je odločil, da je zdravilo Orfadin učinkovita terapija za zdravljenje HT- 1, zlasti če se z njo začne dovolj zgodaj, preden bolnikova jetra utrpijo preveliko škodo, in da zagotavlja boljše rezultate kot zdravljenje samo z dieto, o katerem poroča literatura.
The CHMP decided that Orfadin’ s benefits are greater than its risks and recommended that Orfadin be given marketing authorisation.
CHMP je odločil, da so koristi zdravila Orfadin večje od z njim povezanih tveganj, zato je za zdravilo Orfadin priporočil podelitev dovoljenja za promet z zdravilom.
Orfadin has been authorised under "Exceptional Circumstances", because, as the disease is rare, it has not been possible to obtain complete information on the medicine.
Ker je bolezen redka, ni bilo mogoče pridobiti vseh informacij o zdravilu, zato je dovoljenje za promet z zdravilom Orfadin podeljeno v "izjemnih okoliščinah".
Every year, the European Medicines Agency (EMEA) reviews any new information which may have become available and updates this summary as necessary.
Vsako leto Evropska agencija za zdravila (EMEA) pregleda vse razpoložljive nove informacije in po potrebi posodobi ta povzetek.
What information is still awaited for Orfadin?
Katere informacije o zdravilu Orfadin še pričakujemo?
The company that makes Orfadin has established a post-marketing surveillance program to monitor the use and safety of the medicine in the treatment of HT-1.
Proizvajalec zdravila Orfadin je vzpostavil program postmarketinškega nadzora za spremljanje uporabe in varnosti zdravila pri zdravljenju HT- 1.
Other information about Orfadin:
Druge informacije o zdravilu Orfadin:
The European Commission granted a marketing authorisation valid throughout the European Union for Orfadin to Swedish Orphan International AB on 21 February 2005.
Evropska komisija je dovoljenje za promet z zdravilom Orfadin, veljavno po vsej Evropski uniji, odobrila družbi Swedish Orphan International AB dne 21. februarja 2005.
The record of Orfadin’ s designation as an Orphan medicine is available here.
Zapis o določitvi zdravila Orfadin kot zdravila sirote je na voljo tukaj.
The full EPAR for Orfadin can be found here.
Celotno Evropsko javno poročilo o oceni zdravila (EPAR) za zdravilo Orfadin je na voljo tukaj.
This summary was last updated in 8-2006.
Ta povzetek je bil nazadnje posodobljen 08- 2006.
Hard capsule Hard capsule Hard capsule
Trda kapsula Trda kapsula Trda kapsula
Oral use Oral use Oral use
Peroralno Peroralno Peroralno
Bottle (HDPE) Bottle (HDPE) Bottle (HDPE)
Steklenička (HDPE) Steklenička (HDPE) Steklenička (HDPE)
60 Capsules 60 Capsules 60 Capsules
60 kapsul 60 kapsul 60 kapsul
Orfadin 2 mg hard capsules.
Orfadin 2 mg trde kapsule
Each capsule contains 2 mg nitisinone.
Vsaka kapsula vsebuje 2 mg nitizinona.
White, opaque capsules marked “ NTBC 2mg” on one side of the capsule.
Kapsule motno bele barve z oznako » NTBC 2 mg « na eni strani.
Treatment of patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
Zdravljenje bolnikov s potrjeno diagnozo dedne tirozinemije tipa 1 (HT- 1 – » hereditary tyrosinemia type 1 «), ki so hkrati na dieti z nizko vsebnostjo tirozina in fenilalanina.
Nitisinone treatment should be initiated and supervised by a physician experienced in the treatment of HT-1 patients.
Zdravljenje z nitizinonom mora začeti in nadzorovati zdravnik, usposobljen za zdravljenje bolnikov z HT- 1.
Adjunct to the nitisinone treatment, a diet deficient in phenylalanine and tyrosine is required and should be followed by monitoring of plasma amino acids (see sections 4.4 and 4.8).
Poleg zdravljenja z nitizinonom je potrebna dieta z nizko vsebnostjo fenilalanina in tirozina, pri čemer je treba nadzorovati aminokisline v plazmi (glejte poglavji 4. 4 in 4. 8).
The recommended initial dose is 1 mg/ kg body weight/ day divided in 2 doses administered orally.
Priporočeni začetni odmerek je 1 mg/ kg telesne mase na dan, ki se razdeli v dva odmerka in se jemlje peroralno.
Dose adjustment During regular monitoring, it is appropriate to follow urine succinylacetone, liver function test values and alpha-fetoprotein levels (see section 4.4).
Prilagoditev odmerka Med rednim nadzorovanjem je treba spremljati sukcinilaceton v urinu, teste delovanja jeter in raven alfa- fetoproteina (glejte poglavje 4. 4).
If urine succinylacetone is still detectable one month after the start of nitisinone treatment, the nitisinone dose should be increased to 1.5 mg/ kg body weight/ day divided in 2 doses.
Če en mesec po začetku zdravljenja z nitizinonom v urinu še vedno zasledite sukcinilaceton, je treba odmerek nitizinona povečati na 1, 5 mg/ kg telesne mase na dan in ga razdeliti v dva odmerka.
A dose of 2 mg/ kg body weight/ day may be needed based on the evaluation of all biochemical parameters.
Na podlagi ocene biokemičnih parametrov bo morda potreben odmerek 2 mg/ kg telesne mase na dan.
However, in addition to the tests above, during the initiation of therapy or if there is a deterioration, it may be necessary to follow more closely all available biochemical parameters (i. e. plasma succinylacetone, urine 5-aminolevulinate (ALA) and erythrocyte porphobilinogen (PBG)-synthase activity).
Poleg zgoraj navedenih testov bo med začetkom zdravljenja ali v primeru poslabšanja morda treba podrobneje spremljati vse razpoložljive biokemične parametre (npr. sukcinilaceton v plazmi, 5 - aminolevulinat (ALA) v urinu in delovanje eritrocitne porfobilinogen- sintaze (PBG).
2 Mothers receiving nitisinone should not breast-feed (see sections 4.6 and 5.3).
2 Matere, ki jemljejo nitizinon, ne smejo dojiti (glejte poglavji 4. 6 in 5. 3).
Dietary restriction in relation to visual disorders It is recommended that a slit-lamp examination of the eyes is performed before initiation of nitisinone treatment.
Zdravljenje z dieto v povezavi z motnjami vida Pred začetkom zdravljenja z nitizinonom je priporočljivo opraviti pregled oči s špranjsko svetilko.
It should be established that the patient is adhering to his dietary regimen and the plasma tyrosine concentration should be measured.
Zdravnik mora ugotoviti, ali se bolnik drži dietnega režima, in izmeriti koncentracijo tirozina v plazmi.
A more restricted tyrosine and phenylalanine diet should be implemented in case the plasma tyrosine level is above 500 micromol/ l.
Če raven tirozina v plazmi presega 500 mikromolov/ l, je treba z dieto še bolj zmanjšati vnos tirozina in fenilalanina.
It is not recommended to lower the plasma tyrosine concentration by reduction or discontinuation of nitisinone, since the metabolic defect may result in deterioration of the patient’ s clinical condition.
Koncentracije tirozina v plazmi ni priporočljivo zniževati z zmanjšanjem ali prekinitvijo dajanja nitizinona, ker lahko presnovna motnja poslabša bolnikovo klinično stanje.
Liver monitoring The liver function should be monitored regularly by liver function tests and liver imaging.
Spremljanje jeter Delovanje jeter je treba redno spremljati s testi delovanja jeter in slikanjem.
It is recommended also to monitor serum alpha-fetoprotein concentration.
Priporočljivo je tudi spremljanje koncentracije alfa- fetoproteina v serumu.
Increase in serum alpha- fetoprotein concentration may be a sign of inadequate treatment.
Povečanje koncentracije alfa- fotoproteina je lahko znak neustreznega zdravljenja.
Patients with increasing alpha- fetoprotein or signs of nodules in the liver should always be evaluated for hepatic malignancy.
Pri bolnikih s povečanim alfa- fetoproteinom ali znaki vozlov na jetrih je treba oceniti možnost, da na jetrih nastane maligna tvorba.
Platelet and white blood cell (WBC) monitoring It is recommended that platelet and white cell counts are monitored regularly, as a few cases of reversible trombocytopenia and leucopenia were observed during clinical evaluation.
Spremljanje trombocitov in belih krvnih celic Priporočljivo je redno spremljanje števila trombocitov in belih krvnih celic, ker so med kliničnim ocenjevanjem opazili nekaj primerov reverzibilne trombocitopenije in levkopenije.
Based on in vitro studies, nitisinone is not expected to inhibit CYP 1A2, 2C9, 2C19, 2D6, 2E1 or 3A4- mediated metabolism.
Študije in vitro so pokazale, da nitizonon ne bi smel zavirati presnove, ki deluje preko encimov CYP 1A2, 2C9, 2C19, 2D6, 2E1 ali 3A4.
However, nitisinone has been co- administered with food during the generation of efficacy and safety data.
Formalnih študij interakcij s hrano niso opravili, vendar so med zbiranjem podatkov o učinkovitosti in varnosti nitizinon dajali skupaj s hrano.
Therefore, it is recommended that if nitisinone treatment is initiated with food, this should be maintained on a routine basis.
Če se zdravljenje z nitizinonom začne ob hrani, se priporoča, da se to vzdržuje rutinsko.
Pregnancy There are no adequate data from the use of nitisinone in pregnant women.
Nosečnost Ni zadostnih podatkov o uporabi nitizinona pri nosečnicah.
Nitisinone should not be used during pregnancy unless clearly necessary.
Nitizinona ne smete uporabljati med nosečnostjo, razen če je nujno potrebno.
Lactation It is not known whether nitisinone is excreted in human breast milk.
Dojenje Ni znano, ali se nitizinon izloča v človeško mleko.
Therefore, mothers receiving nitisinone should not breast-feed, since a risk to the suckling child cannot be excluded (see sections 4.3 and 5.3).
Matere, ki jemljejo Orfadin, ne smejo dojiti, ker tveganja za dojenčka ni mogoče izključiti (glejte poglavji 4. 3 in 5. 3).
3 4.8 Undesirable effects
3 4. 8 Neželeni učinki
The adverse reactions considered at least possibly related to treatment are listed below, by body system organ class, and absolute frequency.
Neželene reakcije, ki so vsaj verjetno povezane z zdravljenjem, so navedene spodaj in so razvrščene na podlagi organskih sistemov in absolutne pogostnosti.
Frequencies are defined as common (≥ 1/ 100, < 1/ 10) or uncommon (≥ 1/ 1,000, < 1/ 100).
Pogostnosti so označene kot pogoste (≥1/ 100, < 1/ 10) oziroma občasne (≥1/ 1. 000, < 1/ 100).
Blood and lymphatic system disorders Common: thrombocytopenia, leucopenia, granulocytopenia Uncommon: leukocytosis
Bolezni krvi in limfatičnega sistema Pogoste: trombocitopenija, levkopenija, granulocitopenija Občasne: levkocitoza
Eye disorders Common: conjunctivitis, corneal opacity, keratitis, photophobia, eye pain Uncommon: blepharitis
Očesne bolezni Pogoste: konjunktivitis, motnjava roženice, keratitis, fotofobija, bolečine v očeh Občasne: blefaritis
Skin and subcutaneous tissue disorders Uncommon: exfoliative dermatitis, erythematous rash, pruritus
Bolezni kože in podkožja Občasne: eksfoliativni dermatitis, kožni izpuščaji, pruritus
Nitisinone treatment is associated with elevated tyrosine levels.
Zdravljenje z nitizinonom je povezano s povišano ravnijo tirozina.
Elevated levels of tyrosine have been associated with corneal opacities and hyperkeratotic lesions.
Povečane ravni tirozina so povezane z motnjavo roženice in hiperkeratoznimi lezijami.
Restriction of tyrosine and phenylalanine in the diet should limit the toxicity associated with this type of tyrosinemia (see section 4.4).
Omejitev tirozina in fenilalanina v dieti bi morala omejiti toksičnost, ki je povezana s tem tipom tirozinemije (glejte poglavje 4. 4).
Restriction of tyrosine and phenylalanine in the diet should limit toxicity associated with this type of tyrosinemia.
Zmanjšanje količine tirozina in fenilanalina v dieti bi morale zmanjšati toksičnosti, povezane s to vrsto tirozinemije.
ATC code:
Razna zdravila za bolezni prebavil in presnove Oznaka ATC:
The biochemical defect in hereditary tyrosinemia type 1 (HT-1) is a deficiency of fumarylacetoacetate hydrolase, which is the final enzyme of the tyrosine catabolic pathway.
Biokemična motnja pri dedni tirozinemiji tipa 1 (HT- 1) je pomanjkanje fumarilacetoacetat- hidrolaze, ki je končni encim v katabolni poti tirozina.
Nitisinone is a competitive inhibitor of 4-hydroxyphenylpyruvate dioxygenase, an enzyme which precedes fumarylacetoacetate hydrolase in the tyrosine catabolic pathway.
Nitizinon je kompetitivni inhibitor encima 4 - hidroksifenilpiruvat- dioksigenaze, ki v katabolni poti tirozina katalizira stopnjo pred fumarilacetoacetat- hidrolazo.
By inhibiting the normal catabolism of tyrosine in patients with HT-1, nitisinone prevents the accumulation of the toxic intermediates maleylacetoacetate and fumarylacetoacetate.
Pri inhibiciji normalnega katabolizma tirozina pri bolnikih, ki imajo HT - 1, nitizinon prepreči akumulacijo toksičnih intermediatov, kot sta maleilacetoacetat in fumarilacetoacetat.
In patients with HT-1, these intermediates are converted to the toxic metabolites succinylacetone and succinylacetoacetate.
Pri bolnikih z HT- 1 se ta intermediata pretvorita v toksična presnovka sukcinilaceton in sukcinilacetoacetat.
Succinylacetone inhibits the porphyrin synthesis pathway leading to the accumulation of 5-aminolevulinate.
Sukcinilaceton zavira proces sinteze porfirina in povzroča akumulacijo 5- aminolevulinata.
Nitisinone treatment leads to normalised porphyrin metabolism with normal erythrocyte PBG- synthase activity and urine 5-ALA, decreased urinary excretion of succinylacetone, increased plasma tyrosine concentration and increased urinary excretion of phenolic acids.
Zdravljenje z nitizinonom omogoča, da se vzpostavi normala presnova porfirina z normalnim delovanjem eritrocitne porfobilinogen- sintaze in 5- aminolevulinatom v urinu, da se zmanjša izločanje sukcinilacetona z urinom, poveča koncentracija tirozina v plazmi in tudi odvajanje fenola z urinom.
Available data from a clinical study indicates that in more than 90% of the patients urine succinylacetone was normalized during the first week of treatment.
Razpoložljivi podatki klinične študije so pokazali, da se je sukcinilaceton v urinu pri več kot 90% bolnikov normaliziral v prvem tednu zdravljenja.
4 Effects on overall survival When compared to data for historical controls, it can be seen that treatment with nitisinone together with dietary restriction results in a better survival probability in all HT-1 phenotypes.
4 Učinek na preživetje Pri primerjavi podatkov o pojavnosti bolezni v preteklosti je mogoče opaziti, da se zdravljenje z nitizinonom, ki poteka hkrati z zdravljenjem z dieto, pri vseh fenotipih HT- 1, odraža v večji verjetnosti za preživetje.
Age at start of treatment
Starost na začetku
or diagnosis
zdravljenja ali diagnoze
Nitisinone treatment
Zdravljenje z nitizinona
Dietary control *
Dietna kontrola *
10 years
10 let 5 let
< 2 months > 2-6 months > 6 months
< 2 meseca > 2– 6 mesecev > 6 mesecev * Iz slike 1, Van Spronsen et al.,
82 95 92
82 95 92 1994.
28 51 93
-- 28 95 51 86 93
Treatment with nitisinone was also found to result in reduced risk for the development of hepatocellular carcinoma (2.3 to 3.7-fold) compared to historical data on treatment with dietary restriction alone.
Poleg tega so odkrili, da zdravljenje z nitizinonom, v primerjavi s podatki o zdravljenju zgolj s predpisano dieto, zmanjšuje tveganje za razvoj hepatocelularnega karcinoma (2, 3 - do 3, 7- krat).
It was found that the early initiation of treatment resulted in a further reduced risk for the development of hepatocellular carcinoma (13.5-fold when initiated prior to the age of 12 months).
Odkrili so, da je zgodnji začetek zdravljenja še bolj zmanjšal tveganje za razvoj hepatocelularnega karcinoma (13, 5- krat, če se je zdravljenje začelo pred 12 mesecem starosti).
In 10 healthy male volunteers, after administration of a single dose of nitisinone capsules (1 mg/ kg body weight) the terminal half-life (median) of nitisinone in plasma was 54 hours.
Pri desetih zdravih moških prostovoljcih je bil končni razpolovni čas (mediana) nitizinona v plazmi, po zaužitju enega odmerka kapsul nitizinona (1 mg/ kg telesne mase), 54 ur.
Population pharmacokinetic analysis has been conducted on a group of 207 HT-1 patients.
Populacijske farmakokinetične analize so bile izvedene na skupini 207 bolnikov z TH- 1.
The clearance and half-life were determined to be 0.0956 l/ kg body weight/ day and 52.1 hours respectively.
Očistek zdravila je bil 0, 0956 l/ kg telesne mase na dan, razpolovni čas pa 52, 1 ure.
In the rabbit, nitisinone induced a dose-related increase in malformations (umbilical hernia and gastroschisis) from a dose level of 2.5-fold higher than the maximum recommended human dose (2 mg/ kg/ day).
Pri kuncih je pri odmerkih, večjih od 2, 5- kratnega največjega priporočenega odmerka za človeka (2 mg/ kg/ dan), povzročil od odmerka odvisno povečanje pogostosti malformacij (umbilikalne hernije in gastroshize).
A pre and postnatal development study in the mouse showed statistically significant reduced pup survival and pup growth during the weaning period at dose levels of 125- and 25-fold, respectively, the maximum recommended human dose, with a trend effect on pup survival starting from the dose of 5 mg/ kg/ day.
Trend učinka na preživetje mladičev se začne pri odmerku 5 mg/ kg/ dan.
There was no evidence of in vivo genotoxicity (mouse micronucleus assay and mouse liver UDS assay).
In vivo ni bilo znakov genotoksičnosti (test mikronukleusa pri miših in test o nepravilni sintezi DNK pri miših (USD)).
Carcinogenicity studies have not been performed.
Študij kancerogenosti niso opravili.
Capsule content pregelatinised starch (maize)
Vsebina kapsule predgeliran koruzni škrob
Capsule shell gelatin titanium dioxide (E 171) imprint: black iron oxide (E 172), shellac, propylene glycol
Zunanjost kapsule želatina titanov dioksid (E 171) vtis: črn železov oksid (E 172), šelak, propilenglikol
During the shelf life the patient may store the finished product for a single period of 3 months at a temperature not above 25°C, after which the product must be discarded.
Med rokom uporabnosti lahko bolnik shranjuje zdravilo pri temperaturi do 25 °C le 3 mesece, potem pa ga mora zavreči.
High density polyethylene bottle with a tamper proof low density polyethylene cap, containing 60 capsules.
Steklenička iz gostega polietilena z neprepustno zaporko in redkega polietilena vsebuje 60 kapsul.
Orfadin 5 mg hard capsules.
Orfadin 5 mg trde kapsule
Each capsule contains 5 mg nitisinone.
Vsaka kapsula vsebuje 5 mg nitizinona.
White, opaque capsules marked “ NTBC 5mg” on one side of the capsule.
Kapsule motno bele barve z oznako » NTBC 5 mg « na eni strani.
However, in addition to the tests above, during the initiation of therapy or if there is a deterioration it may be necessary to follow more closely all available biochemical parameters (i. e. plasma succinylacetone, urine 5-aminolevulinate (ALA) and erythrocyte porphobilinogen (PBG)-synthase activity).
Poleg zgoraj navedenih testov bo med začetkom zdravljenja ali v primeru poslabšanja morda treba podrobneje spremljati vse razpoložljive biokemične parametre (npr. sukcinilaceton v plazmi, 5 - aminolevulinat (ALA) v urinu in delovanje eritrocitne porfobilinogen- sintaze (PBG).
7 4.3 Contraindications
4. 3 Kontraindikacije
Mothers receiving nitisinone should not breast-feed (see sections 4.6 and 5.3).
7 Matere, ki jemljejo nitizinon, ne smejo dojiti (glejte poglavji 4. 6 in 5. 3).
8 4.7 Effects on ability to drive and use machines
4. 7 Vpliv na sposobnost vožnje in upravljanja s stroji
Blood and lymphatic system disorders Common: thrombocytopenia, leucopenia, granulocytopenia Uncommon: leucocytosis
Bolezni krvi in limfatičnega sistema Pogoste: trombocitopenija, levkopenija, granulocitopenija Občasne: levkocitoza
Available data from a clinical study indicates that in more than 90% of the patients urine succinylacetone was normalized during the
Razpoložljivi podatki klinične študije so pokazali, da se je sukcinilaceton v urinu pri več kot 90% bolnikov normaliziral v prvem tednu zdravljenja.
Effects on overall survival When compared to data for historical controls, it can be seen that treatment with nitisinone together with dietary restriction results in a better survival probability in all HT-1 phenotypes.
9 Učinek na preživetje Pri primerjavi podatkov o pojavnosti bolezni v preteklosti je mogoče opaziti, da se zdravljenje z nitizinonom, ki poteka hkrati z zdravljenjem z dieto, pri vseh fenotipih HT- 1, odraža v večji verjetnosti za preživetje.
In vitro studies using human liver microsomes and cDNA-expressed P450 enzymes have shown limited CYP 3A4-mediated metabolism.
Študije in vitro, pri katerih so uporabili človeške mikrosome jeter in s cDNA izražene encime P450, so pokazale omejeno presnovo preko encima CYP 3A4.
Orfadin 10 mg hard capsules.
Orfadin 10 mg trde kapsule
Each capsule contains 10 mg nitisinone.
Vsaka kapsula vsebuje 10 mg nitizinona.
White, opaque capsules marked “ NTBC 10mg” on one side of the capsule.
Kapsule motno bele barve z oznako » NTBC 10 mg « na eni strani.