natgillin/translations-raw huggingface dataset:
There was no need for ‘bioequivalence' studies to investigate whether Pazenir is absorbed similarly to the reference medicine to produce the same level of the active substance in the blood.
Izvedba študije „bioekvivalence“, s katero bi raziskali, ali se zdravilo Pazenir absorbira podobno kot referenčno zdravilo in tako doseže enako raven učinkovine v krvi, ni bila potrebna.
Pazenir is given as an infusion into a vein over a period of 30 minutes.
Zdravilo Pazenir se daje z infundiranjem v veno, ki traja 30 minut.
Pazenir should only be given under the supervision of a cancer doctor in clinics that are specialised in giving ‘cytotoxic' (cell-killing) medicines.
Zdravilo Pazenir se lahko daje samo pod nadzorom zdravnika specialista za rakava obolenja v bolnišnicah, ki so specializirana za dajanje citotoksičnih zdravil (zdravil, ki uničujejo celice).
In Pazenir, as in its reference medicine Abraxane, paclitaxel is attached to a human protein called albumin in tiny particles known as ‘nanoparticles'.
V zdravilu Pazenir (enako kot v njegovem referenčnemu zdravilu Abraxane) je paklitaksel vezan na človeško beljakovino albumin v drobnih delcih, imenovanih nanodelci.
• metastatic adenocarcinoma of the pancreas, as a first treatment in combination with another cancer medicine, gemcitabine;
• metastatskega adenokarcinoma trebušne slinavke, pri katerem se uporablja kot zdravilo prve izbire v kombinaciji z drugim zdravilom za zdravljenje raka, tj. gemcitabinom;
The active substance in Pazenir, paclitaxel, belongs to the group of cancer medicines known as the ‘taxanes'.
Učinkovina v zdravilu Pazenir, paklitaksel, spada v skupino zdravil za zdravljenje raka, imenovanih taksani.
Pazenir contains the active substance paclitaxel attached to a human protein called albumin and is a ‘generic medicine'.
Zdravilo Pazenir vsebuje učinkovino paklitaksel, vezano na človeško beljakovino, imenovano albumin, in je generično zdravilo.
• non-small cell lung cancer, as a first treatment in combination with the cancer medicine carboplatin when the patient cannot have surgery or radiotherapy.
• nedrobnoceličnega pljučnega raka, pri katerem se uporablja kot zdravilo prve izbire v kombinaciji z zdravilom za zdravljenje raka, imenovanim karboplatin, pri bolnikih, pri katerih kirurški poseg ali obsevanje nista primerna.
In metastatic breast cancer, Pazenir is given on its own every three weeks.
Pri metastatskem raku dojk se zdravilo Pazenir vsake tri tedne daje kot samostojno zdravilo.
In metastatic adenocarcinoma of the pancreas, Pazenir is given in 4-week treatment cycles.
Pri metastatskem adenokarcinomu trebušne slinavke se zdravilo Abraxane daje v štiritedenskih ciklih zdravljenja.
How has Pazenir been studied?
Kako je bilo zdravilo Pazenir raziskano?
Therefore, the Agency's view was that, as for Abraxane, the benefits of Pazenir outweigh the identified risks and it can be authorised for use in the EU.
Menila je, da koristi zdravila Pazenir enako kot pri zdravilu Abraxane odtehtajo znana tveganja in da se zdravilo lahko odobri za uporabo v EU.
How is Pazenir used?
Kako se zdravilo Pazenir uporablja?
How does Pazenir work?
Kako zdravilo Pazenir deluje?
Rash and Severe Cutaneous Adverse Reactions safety data from Phases 2 and 3 Incidence of rash and severe cutaneous reactions Grading and management of rash and severe cutaneous reactions, particularly with respect to criteria for the continuation or discontinuation of telaprevir and the other
Navodilo za uporabo za zdravnike mora vsebovati naslednje ključne elemente: podatke o varnosti iz študij fazeIIinIII o izpuščaju in hudih kožnih reakcijah pojavnost izpuščaja in hudih kožnih reakcij merila za oceno in zdravljenje izpuščaja in hudih kožnih reakcij, še posebno kriterije za nadaljevanje ali prekinitev zdravljenja s telaprevirjem in drugimizdravili slike izpuščajev različnih stopenj
The Member States shall ensure that the MAH provides to all physicians who are expected to prescribe or use INCIVO a healthcare professional educational pack containing the following:
Imetnik dovoljenja za promet z zdravilom bo zagotovil, da bodo zdravniki, ki bodo predpisovali ali uporabljali zdravilo INCIVO prejeli izobraževalno gradivo, ki bo vsebovalo: Povzetek glavnih značilnosti zdravila
The Member States should ensure that all conditions or restrictions with regard to the safe and effective use of the medicinal product described below are implemented:
DODATEK POGOJI IN OMEJITVE GLEDE VARNE IN UČINKOVITE UPORABE ZDRAVILA
The Member States shall agree to the final healthcare educational pack with the Marketing Authorization Holder (MAH) prior to launch of the product in their territory.
Imetnik dovoljenja za promet z zdravilom se bo za obliko in vsebino izobraževalnih gradiv za zdravstvene delavce pred dajanjem zdravila na trg dogovoril s pristojnim nacionalnim organom države članice.
The Summary of Product Characteristics
Navodilo za uporabo za bolnike
250 µg Solution for injection
250 µg Raztopina za injiciranje
5 capsules
5 kapsul
Intravenous use vial (glass) 5 ml
Intravensko Viala (steklo)
Packaging Content (concentration) Package size
Izmišljeno ime jakost farmacevtska oblika pot uporabe ovojnina vsebina velikost pakiranja
If you are in a compassionate use programme and need more information about your treatment, contact the doctor who is giving it to you.
Če ste vključeni v program sočutne uporabe in potrebujete več informacij o zdravljenju, se obrnite na zdravnika, ki vam je predpisal zdravljenje.
The conditions are caused by a lack of enzymes needed to remove them.
Bolezni sta posledica pomanjkanja encimov, ki so potrebni za odstranjevanje navedenih snovi.
In its letter notifying the Agency of the withdrawal of the application, the company stated that it needed more time to prepare adequate answers to questions raised by the CHMP.
Družba je v dopisu, s katerim je agencijo obvestila o umiku vloge, navedla, da potrebuje več časa za pripravo ustreznih odgovorov na vprašanja, ki jih je zastavil odbor CHMP.
Prohippur was not studied directly in patients with these conditions.
Zdravila Prohippur niso proučevali neposredno pri bolnikih s temi boleznimi.
What consequences does this withdrawal have for patients in clinical trials or compassionate use programmes?
Kakšne so posledice umika vloge za bolnike, ki sodelujejo v kliničnih preskušanjih ali programih sočutne uporabe zdravila?
It was to be available as granules.
Na voljo naj bi bilo v obliki zrnc.
Prohippur is a medicine containing the active substance sodium benzoate.
Prohippur je zdravilo, ki vsebuje učinkovino natrijev benzoat.
Finally, the CHMP had questions about the high levels of a substance in Prohippur (butylated methacrylate copolymer), the method of administration and quality control of the medicine in general.
Na koncu je imel odbor CHMP tudi vprašanja o visokih ravneh snovi v zdravilu Prohippur (kopolimera butiliranega metakrilata), načinu dajanja zdravila in nadzoru kakovosti zdravila na splošno.
The application was withdrawn after the CHMP had evaluated the documentation provided by the company and formulated lists of questions.
Vlogo so umaknili po tem, ko je odbor CHMP že ocenil dokumentacijo, ki jo je predložila družba, in oblikoval seznam vprašanj.
The company informed the CHMP that there are no ongoing clinical trials with Prohippur and that a compassionate use programme approved in France will continue as planned.
Družba je odbor CHMP obvestila, da trenutno ne potekajo klinična preskušanja zdravila Prohippur in da se bo program sočutne uporabe, odobren v Franciji, nadaljeval po načrtu.
Prohippur was expected to be used to treat the following inherited disorders:
Zdravilo Prohippur naj bi se uporabljalo za zdravljenje naslednjih dednih bolezni:
Prohippur was designated an ‘orphan medicine' (a medicine to be used in rare diseases) for non-ketotic hyperglycinaemia on 11 September 2002 and for urea cycle disorders on 14 July 2016, 29 August 2016 and 18 November 2016.
Zdravilo Prohippur je bilo 11. septembra 2002 določeno kot „zdravilo sirota“ (zdravilo za zdravljenje redkih bolezni) za zdravljenje neketotične hiperglicinemije ter 14. julija 2016, 29. avgusta 2016 in 18. novembra 2016 za zdravljenje motenj ciklusa sečnine.
Both glycine and ammonia are harmful in high amounts and can cause severe brain damage.
Glicin in amoniak sta v večjih količinah škodljiva in lahko povzročata hude možganske poškodbe.
The company had not responded to the last round of questions at the time of the withdrawal.
Družba ob umiku vloge še ni odgovorila na zadnji sklop vprašanj.
Prohippur also helps remove excess nitrogen from the body, thereby lowering levels of ammonia.
Zdravilo Prohippur prav tako pomaga odstraniti odvečni dušik iz telesa in tako zniža raven amoniaka.
The CHMP noted that the published studies submitted by the company did not provide enough evidence of the effectiveness and safety of sodium benzoate in the various disorders.
Ugotovil je, da objavljene študije, ki jih je predložila družba, ne zagotavljajo dovolj dokazov o učinkovitosti in varnosti natrijevega benzoata pri različnih boleznih.
On 3 April 2018, Lucane Pharma officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wished to withdraw its application for a marketing authorisation for Prohippur, for the treatment of non-ketotic hyperglycinaemia and urea cycle disorders.
Družba Lucane Pharma je 3. aprila 2018 uradno obvestila Odbor za zdravila za uporabo v humani medicini (CHMP), da želi umakniti vlogo za pridobitev dovoljenja za promet z zdravilom Prohippur, namenjenim zdravljenju neketotične hiperglicinemije in motenj ciklusa sečnine.
Furthermore, as Prohippur was not used in clinical trials, the company should have provided data to show that Prohippur granules worked in the same way as the sodium benzoate used in the published studies.
Ker se zdravilo Prohippur ni uporabljalo v kliničnih preskušanjih, bi morala družba predložiti podatke, ki kažejo, da zrnca zdravila Prohippur delujejo enako kot natrijev benzoat, ki so ga uporabljali v objavljenih študijah.
The active substance in Prohippur, sodium benzoate, helps reduce harmful levels of glycine by attaching to glycine and forming a substance that is more easily removed by the kidneys.
Učinkovina zdravila Prohippur, natrijev benzoat, pomaga znižati škodljive ravni glicina, tako da se pritrdi na glicin in tvori snov, ki jo ledvice lažje odstranijo.
What was Prohippur expected to be used for?
Za kaj naj bi se zdravilo Prohippur uporabljalo?
The company presented data from published studies on the use of sodium benzoate in patients with non-ketotic hyperglycinaemia and urea cycle disorders.
Družba je predložila podatke objavljenih študij o uporabi natrijevega benzoata pri bolnikih z neketotično hiperglicinemijo in motnjami ciklusa sečnine.
Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Prohippur did not outweigh its risks.
Zato je ob umiku vloge menil, da koristi zdravila Prohippur ne odtehtajo z njim povezanih tveganj.
• non-ketotic hyperglycinaemia, where abnormally high levels of the amino acid glycine occur in the blood.
• neketotične hiperglicinemije, pri kateri se v krvi pojavljajo nenormalno visoke ravni aminokisline glicina;
• urea cycle disorders, a group of conditions where abnormally high levels of nitrogen occur in the body in the form of ammonia.
• motenj ciklusa sečnine, tj. skupine stanj, pri katerih se v telesu pojavljajo nenormalno visoke ravni dušika v obliki amoniaka.
30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom27 April 2018 EMA/258015/2018 EMEA/H/C/004150
EMA/258015/2018 EMEA/H/C/004150
At the time of the withdrawal, the CHMP had some concerns.
Odbor CHMP je ob umiku vloge izrazil določene zadržke.
What is Prohippur?
Kaj je zdravilo Prohippur?
How does Prohippur work?
Kako zdravilo Prohippur deluje?
5 years.
5 leta
Date of first authorisation: 20 November 2002 Date of latest renewal: 20 November 2012
Datum prve odobritve: 20. november 2002 Datum zadnjega podaljšanja: 20. november 2012
A relevance of these findings to humans cannot be excluded.
Pomembnost teh ugotovitev za človeka ni mogoče izključiti.
You should swallow the whole capsule with a glass of water.
Kapsulo morate pogoltniti cele s kozarcem vode.
The other ingredients are:
Druge sestavine zdravila so:
Recommended dose 200 mg three times a day 200 mg twice a day 100 mg three times a day 100 mg twice a day 100 mg once a day
200 mg trikrat dnevno 200 mg dvakrat dnevno 100 mg trikrat dnevno 100 mg dvakrat dnevno 100 mg enkrat dnevno
If you have a problem with your kidneys you may receive a lower starting dose.
Če imate težave s svojimi ledvicami, lahko dobite manjši začetni odmerek.
- if you are allergic to miglustat or any of the other ingredients of this medicine (listed in section 6)
- če ste alergični na miglustat ali katero koli sestavino tega zdravila (navedeno v poglavju 6).
Maximum plasma concentrations are reached about 2 hours after dose.
Najvišje koncentracije v plazmi dosega približno 2 uri po odmerku.
Weight loss has been reported in 55% of patients.
O izgubi telesne mase so poročali pri približno 55 % bolnikov.
Do not take this medicine after the expiry date which is stated on the carton after ‘EXP'.
Tega zdravila ne smete uporabljati po datumu izteka roka uporabnosti, ki je naveden na škatli poleg oznake »EXP«.
• Risk Management Plan (RMP)
ZDRAVILA • Načrt za obvladovanje tveganj (RMP)
A No Observed Effect Level (NOEL) was not established and the effect was not dose dependent.
Ravni brez opaženega učinka (NOEL - ˝No Observed Effect Level˝) niso ugotovili, učinek ni bil odvisen od odmerka.
This leaflet was last revised in:
Navodilo je bilo nazadnje revidirano dne:
The tremor often disappears without needing to stop the treatment.
Tremor pogosto izgine brez potrebe prekinitve zdravljenja z zdravilom Zavesca.
What Zavesca is and what it is used for
Kaj je zdravilo Zavesca in za kaj ga uporabljamo
Capsule, hard 84 capsules
kapsula, trda 84 kapsul
Patients with chronic diarrhoea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice.
Bolnike s kronično diarejo ali drugimi trdovratnimi prebavnimi težavami, ki se na te ukrepe ne odzovejo, je treba preiskati skladno s klinično prakso.
Zavesca can be taken with or without food.
Zdravilo Zavesca se lahko jemlje s hrano ali brez nje.
Most serious side effects:
Najresnejši neželeni učinki:
The tests will help the doctor decide whether these effects are due to your disease or other existing conditions, or due to side effects of Zavesca (see section 4 for further details).
Te preiskave bodo zdravniku v pomoč pri presoji, ali so ti učinki posledica vaše bolezni ali drugega obstoječega stanja ali posledica neželenih učinkov zdravila Zavesca (za dodatne informacije glejte poglavje 4).
What Zavesca is and what it is used for What you need to know before you take Zavesca How to take Zavesca
Kaj je zdravilo Zavesca in za kaj ga uporabljamo Kaj morate vedeti, preden boste vzeli zdravilo Zavesca Kako jemati zdravilo Zavesca
Concomitant administration of food decreases the rate of absorption (Cmax was decreased by 36% and tmax delayed 2 hours), but has no statistically significant effect on the extent of absorption of miglustat (AUC decreased by 14%).
Sočasno jemanje s hrano zniža raven absorpcije (Cmax se je znižal za 36 % in tmax podaljšal za 2 uri), vendar nima statistično značilnega učinka na absorpcijo miglustata (AUC se je znižala za 14 %).
If you are pregnant, breast feeding, think you may be pregnant or planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.
Če ste noseči ali dojite, menite, da bi lahko bili noseči ali načrtujete zanositev, se posvetujte z zdravnikom ali farmacevtom, preden vzamete to zdravilo.
Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Zavesca.
Obvestite zdravnika, če jemljete zdravila, ki vsebujejo imiglucerazo, ki se včasih jemljejo skupaj z zdravilom Zavesca.
They may lower the amount of Zavesca in your body.
Ta zdravila lahko znižajo količino zdravila Zavesca v vašem telesu.
It is not known if miglustat is secreted in breast milk.
Ni znano, ali se miglustat izloča v materino mleko.
Among the 41 patients enrolled in the trial 14 patients were treated with Zavesca for more than 3 years.
Med 41 bolniki, vključenimi v preskušanje, je bilo 14 bolnikov zdravljenih z zdravilom Zavesca več kot 3 leta.
Do not give this medicine to children and adolescents (below 18 years old) with type 1 Gaucher disease because it is not known if it works in this disease.
Tega zdravila ne dajajte otrokom in mladostnikom (starim manj kot 18 let) z Gaucherjevo boleznijo tipa 1, saj njegov učinek na to bolezen ni znan.
Don't stop taking Zavesca without talking to your doctor.
Ne prenehajte uporabljati zdravila Zavesca, ne da se o tem pogovorite z zdravnikom.
The efficacy and safety of Zavesca has not been specifically evaluated in patients with severe Gaucher disease.
Učinkovitost in varnost zdravila Zavesca nista bili specifično ocenjeni pri bolnikih s hudo Gaucherjevo boleznijo.
Other medicines and Zavesca
Druga zdravila in zdravilo Zavesca
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines
Obvestite zdravnika, če jemljete, ste pred kratkim jemali ali pa boste morda začeli jemati katero koli drugo zdravilo.
Carcinomas in the large intestine occurred occasionally at all doses with a statistically significant increase in the high dose group.
Karcinomi debelega črevesa so se pojavili občasno pri vseh odmerkih, s statistično signifikantnim povečanjem pri največjem odmerku.
Your doctor will perform the following tests before treatment and during treatment with Zavesca: - an examination to check the nerves in your arms and legs - measurement of vitamin B12 levels - monitoring growth if you are a child or adolescent with Niemann-Pick type C disease - monitoring of blood platelet counts
Pred in med zdravljenjem z zdravilom Zavesca bo vaš zdravnik opravil naslednje preiskave: - pregled živcev v vaših rokah in nogah - meritev ravni vitamina B12 - nadziranje rasti, če ste otrok ali mladostnik z Niemann-Pickovo boleznijo tipa C - nadziranje števila trombocitov v krvi
Management In case of overdose general medical care is recommended.
Pri prevelikem odmerjanju se priporoča splošna zdravstvena nega.
Following administration of a single dose of 100 mg 14C-miglustat to healthy volunteers, 83% of the radioactivity was recovered in urine and 12% in faeces.
Po uporabi enega odmerka 100 mg 14C-miglustata pri zdravih prostovoljcih je bilo 83 % radioaktivno označenega zdravila izločenega z urinom in 12 % z blatom.
There are no pharmacokinetic data available in patients with liver impairment or in the elderly (> 70 years).
Na voljo ni nobenih farmakokinetičnih podatkov za bolnike z okvarjenim delovanjem jeter ali starostnike (> 70 let).
This study was conducted over a 6-month randomised comparison period followed by 18 months extension where all patients received Zavesca monotherapy.
To študijo so izpeljali prek 6-mesečnega randomiziranega primerjalnega obdobja, ki mu je sledilo 18-mesečno podaljšanje, v katerem so vsi bolniki prejemali monoterapijo zdravila Zavesca.
This study also indicated that Zavesca has no or limited effect on the pharmacokinetics of imiglucerase.
Ta študija je tudi pokazala, da zdravilo Zavesca nima ali ima le majhen učinek na farmakokinetiko imigluceraze.
Data to support safety and efficacy of Zavesca in Niemann-Pick type C disease come from a prospective open-label clinical trial and a retrospective survey.
Podatki, ki podpirajo varnost in učinkovitost zdravila Zavesca pri Niemann-Pickovi bolezni tipa C so bili zbrani v prospektivnem odprtem kliničnem preskušanju in v retrospektivni analizi.
Your doctor may reduce your dose, e.g., to one capsule (100 mg) once or twice a day, if you suffer from diarrhoea when taking Zavesca (see section 4).
Vaš zdravnik lahko zmanjša vaš odmerek, npr. na eno kapsulo (100 mg) enkrat ali dvakrat dnevno, če imate med jemanjem zdravila Zavesca drisko (glejte poglavje 4).
If you take more capsules than you were told to, consult your doctor immediately.
Če ste vzeli večji odmerek zdravila Zavesca, kot bi smeli Če ste vzeli več kapsul, kot vam je bilo naročeno, se takoj posvetujte z zdravnikom.
If you stop taking Zavesca
Če ste prenehali jemati zdravilo Zavesca
The efficacy of Zavesca in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established.
Učinkovitost zdravila Zavesca pri otrocih in mladostnikih, starih od 0 do 17 let, z Gaucherjevo boleznijo tipa 1, še ni bila dokazana.
Overall the data show that treatment with Zavesca can reduce the progression of clinically relevant neurological symptoms in patients with Niemann-Pick type C disease.
Na splošno so podatki pokazali, da lahko zdravljenje z zdravilom Zavesca zmanjša napredovanje klinično značilnih nevroloških simptomov pri bolnikih z Niemann-Pickovo boleznijo tipa C.
If you have diarrhoea, your doctor may ask you to change your diet to reduce your lactose and carbohydrate intake such as sucrose (cane sugar), or not to take Zavesca together with food, or to temporarily reduce your dose.
Če boste imeli drisko, vam bo morda zdravnik svetoval, da spremenite svojo prehrano tako, da zmanjšate vnos laktoze in ogljikovih hidratov, kot je saharoza (namizni sladkor), ali ne jemljete zdravila Zavesca skupaj s hrano ali začasno znižate svoj odmerek.
In a pooled analysis of uncontrolled data, bone mineral density Z-scores at the lumbar spine and femoral neck increased by more than 0.1 units from baseline in 27 (57%) and 28 (65%) of the patients with longitudinal bone density measurements.
V zbrani analizi nekontroliranih podatkov so se vrednosti Z mineralne gostote kosti ledvene hrbtenice in vratu stegnenice povečale za več kot 0,1 enoto od izhodišča pri 27 (57 %) in 28 (65 %) bolnikov pri vzdolžni smeri merjenja gostote kosti.
The reason for these tests is that some patients have had tingling or numbness in the hands and feet, or a decrease in body weight, while taking Zavesca.
Razlog za te preiskave je, da so nekateri bolniki med jemanjem zdravila Zavesca izkusili omrtvičenost ali mravljinčenje v rokah in stopalih ali zmanjšanje telesne mase.
Approximately 37% of patients in clinical trials in type 1 Gaucher disease, and 58% of patients in a clinical trial in Niemann-Pick type C disease reported tremor on treatment.
Tremor Približno 37 % bolnikov v kliničnih preskušanjih z Gaucherjevo boleznijo tipa 1 in 58 % bolnikov v kliničnih preskušanjih z Niemann-Pickovo boleznijo tipa C je poročalo o tremorju med zdravljenjem.
The main effects common to all species were weight loss and diarrhoea, and, at higher doses, damage to the gastrointestinal mucosa (erosions and ulceration).
Glavni učinki, skupni vsem vrstam, so bili hujšanje in diareja ter, pri višjih odmerkih, poškodbe sluznice prebavil (erozije in ulceracije).
Eighteen patients then continued to receive Zavesca under an optional extended treatment protocol.
18 bolnikov je nato nadaljevalo z jemanjem zdravila Zavesca v neobveznem podaljšanem programu zdravljenja.
Miglustat affected embryo/foetal survival in rats and rabbits, dystocia was reported, post-implantation losses were increased, and an increased incidence of vascular anomalies occurred in rabbits.
Miglustat je vplival na preživetje zarodkov/plodov pri podganah in zajcih, poročali so o distociji, povečale so se izgube po implantaciji in povečala se je incidenca žilnih nepravilnosti pri zajcih.
An open-label, non comparative, 2-year study enrolled 42 patients with type 1 Gaucher disease, who had received a minimum of 3 years of ERT and who fulfilled criteria of stable disease for at least 2 years.
Odprta, neprimerjalna, 2-letna študija z vključenimi 42 bolniki z Gaucherjevo boleznijo tipa 1, ki so vsaj 3 leta prejemali ERT in so izpolnjevali kriterije bolezni, stabilne vsaj 2 leti.