natgillin/translations-raw huggingface dataset:
During the infusion with this higher dose, fever of mild intensity and short duration (5 hours) was observed in one patient.
Tijekom infuzije ove više doze u jednog je bolesnika opažena vrućica blagog intenziteta i kratkog trajanja (5 sati).
Velmanase alfa exhibited a linear (i.e. first-order) pharmacokinetic profile, and Cmax and AUC increased proportionally to the dose with doses ranging from 0.8 to 3.2 mg/kg (corresponding to 25 and 100 units/kg).
Velmanaza alfa pokazala je linearan farmakokinetički profil (tj. prvog reda), a Cmax i AUC povećavali su se proporcionalno dozi u rasponu doza od 0,8 do 3,2 mg/kg (što odgovara 25, odnosno 100 jedinica/kg).
In both cases the patients recovered without sequelae.
U oba slučaja bolesnici su se oporavili bez posljedica.
If the volume of the solution exceeds one syringe capacity, the required number of syringes should be prepared in order to replace the syringe quickly during the infusion.
Ako je volumen otopine veći od kapaciteta štrcaljke, potrebno je pripremiti potreban broj štrcaljki kako bi se štrcaljka mogla brzo zamijeniti tijekom infuzije.
If you experience an infusion-related reaction, you must tell your doctor immediately.
Ako osjetite reakciju povezanu s infuzijom, odmah morate obavijestiti svog liječnika.
The European Medicines Agency has deferred the obligation to submit the results of studies with Lamzede in one or more subsets of the paediatric population in the treatment of alpha-mannosidosis disease (see section 4.2 for information on paediatric use).
Europska agencija za lijekove odgodila je obvezu podnošenja rezultata ispitivanja lijeka Lamzede u jednoj ili više podskupina pedijatrijske populacije u liječenju bolesti alfa-manozidoze (vidjeti dio 4.2 za informacije o pedijatrijskoj primjeni).
This medicine is only to be used under the supervision of a doctor experienced in the treatment of alpha-mannosidosis or other similar diseases and should only be given by a healthcare professional.
Ovaj se lijek smije primjenjivati samo pod nadzorom liječnika s iskustvom u liječenju alfa-manozidoze ili drugih sličnih bolesti i smije ga davati samo zdravstveni radnik.
Acute renal failure was of moderate severity leading to temporary discontinuation of the study treatment and fully resolved within 3 months.
Akutno zatajenje bubrega bilo je umjerene težine i dovelo je do privremenog prekida ispitivanog liječenja te se potpuno povuklo unutar 3 mjeseca.
What you need to know before Lamzede is used Lamzede must not be used:
Što morate znati prije nego počnete primjenjivati Lamzede Nemojte primjenjivati Lamzede:
- If the infusion-related reactions are severe and/or there is a loss of effect from this medicine, your doctor will perform a blood test to check for antibodies that might affect the outcome of your treatment.
- Ako su reakcije povezane s infuzijom teške i/ili se izgubio učinak lijeka, liječnik će provesti krvnu pretragu kako bi provjerio jesu li prisutna protutijela koja bi mogla utjecati na ishod liječenja.
A total of 2 serious adverse reactions (loss of consciousness in 1 patient and acute renal failure in 1 patient) were observed.
Ukupno su opažene 2 ozbiljne nuspojave (gubitak svijesti u 1 bolesnika i akutno zatajenje bubrega u 1 bolesnika).
Serum oligosaccharide concentration (μmol/l) Overall(1)
Koncentracija oligosaharida u serumu (μmol/l) Ukupno(1)
p-value [95% CI]
p-vrijednost [95% CI]
Hypersensitivity reactions have been reported in patients in clinical studies.
Reakcije preosjetljivosti zabilježene su u bolesnika u kliničkim ispitivanjima.
Velmanase alfa vs. placebo
Velmanaza alfa naspram placeba
In the most severe cases, the current medical standards for emergency treatment are to be observed.
U najtežim slučajevima, potrebno je provesti liječenje hitnog stanja prema standardima suvremene medicinske prakse.
This being an approval under exceptional circumstances and pursuant to Article 14(8) of Regulation (EC) No 726/2004, the MAH shall conduct, within the stated timeframe, the following measures:
Uredbe (EZ) br. 726/2004, nositelj odobrenja dužan je unutar navedenog vremenskog roka provesti sljedeće mjere:
Pharmacotherapeutic group: Other alimentary tract and metabolism products, enzymes.
Farmakoterapijska skupina: drugi lijekovi za probavni sustav i metabolizam, enzimi.
- If the infusion-related reaction is severe, your doctor will stop the infusion immediately and start giving you appropriate medical treatment.
Ako je reakcija povezana s infuzijom teška, liječnik će odmah zaustaviti infuziju i početi Vam davati odgovarajuću terapiju lijekovima.
Lamzede is supplied in a vial as a powder for infusion which will be made up with water for injections before being given.
Lamzede se isporučuje u bočici kao prašak za infuziju koji će se prije davanja pripremiti s vodom za injekcije.
If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user and would normally not be longer than 24 hours at 2°C to 8°C.
Ako se ne primijeni odmah, vrijeme proteklo do primjene i uvjeti čuvanja rekonstituiranog lijeka za to vrijeme odgovornost su korisnika i to obično ne bi smjelo biti dulje od 24 sata na temperaturi od 2°C do 8°C.
common (≥1/100 to <1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare
Učestalost je definirana kao vrlo često (≥ 1/10), često (≥ 1/100 i < 1/10), manje često (≥ 1/1000 i < 1/100), rijetko
Infusion-related reaction
Reakcije povezane s infuzijom
For patients weighing less than 18 kg, and receiving less than 9 mL reconstituted solution, the infusion rate should be calculated so that the infusion time is ≥50 minutes.
Za bolesnike tjelesne težine manje od 18 kg koji primaju manje od 9 ml rekonstituirane otopine, potrebno je izračunati brzinu infuzije tako da infuzija traje ≥ 50 minuta.
The recommended dose of Lamzede is 1 mg/kg of body weight given once every week.
Preporučena doza lijeka Lamzede je 1 mg/kg tjelesne težine, primijenjena jedanput svakog tjedna.
Instructions for reconstitution and administration
Upute za rekonstituciju i primjenu
If severe allergic or anaphylactic-type reactions occur, immediate discontinuation of velmanase alfa is recommended and current medical standards for emergency treatment are to be followed.
Ako se jave teške alergijske ili anafilaktičke reakcije, preporučuje se odmah prekinuti primjenu velmanaze alfa i provesti liječenje hitnog stanja prema standardima suvremene medicinske prakse.
There were no apparent pharmacokinetic gender differences in patients with alpha-mannosidosis disease.
Nije bilo vidljivih razlika u farmakokinetici između muških i ženskih bolesnika s bolešću alfa-manozidozom.
The recommended dose regimen is 1 mg/kg of body weight administered once every week by intravenous infusion at a controlled speed.
Preporučeni režim doziranja je 1 mg/kg tjelesne težine primijenjen jedanput tjedno intravenskom infuzijom kontrolirane brzine.
After reconstitution, the medicine should be used immediately.
Nakon rekonstitucije, lijek se mora primijeniti odmah.
The adverse reactions reflecting exposure of 33 patients treated with velmanase alfa in clinical studies are listed in the table 1 below.
Nuspojave koje odražavaju izloženost 33 bolesnika liječena velmanazom alfa u kliničkim ispitivanjima navedene su niže u tablici 1.
The patient received saline infusion in a hospital setting and was then discharged after 6-hour observation.
Bolesnik je primio infuziju fiziološke otopine u bolničkom okruženju te je bio otpušten nakon 6 sati promatranja.
Serum oligosaccharide concentration (µmol/L)
Koncentracija oligosaharida u serumu (µmol/l)
Last observation % change from baseline (SD)
% promjene od početne vrijednosti kod zadnjeg opažanja (SD)
IRRs (including hypersensitivity, nausea, vomiting, pyrexia, chills, feeling hot, malaise, urticaria, anaphylactoid reaction and hyperhidrosis) were reported in 9% of the patients (3 out of 33 patients) in clinical studies.
Reakcije povezane s infuzijom (uključujući preosjetljivost, mučninu, povraćanje, pireksiju, zimicu, osjećaj vrućine, malaksalost, urtikariju, anafilaktoidnu reakciju i hiperhidrozu) bile su zabilježene u 9% bolesnika (3 od 33 bolesnika) u kliničkim ispitivanjima.
The maximum infusion rate is 25 mL/hour.
Najveća brzina infuzije je 25 ml/sat.
Baseline actual value Mean (SD)
Srednja vrijednost stvarne početne vrijednosti (SD)
In order to obtain long-term data on effectiveness and safety of treatment with Lamzede and to characterize the entire alpha-mannosidosis population, including variability of clinical manifestation, progression and natural history, the MAH is requested to submit the results of a study based on adequate source of data deriving from a registry of patients with alpha-mannosidosis.
Kako bi se prikupili dugoročni podaci o učinkovitosti i sigurnosti liječenja lijekom Lamzede te opisala cijela populacija s alfa-manozidozom, uključujući raznolikost kliničkih manifestacija, progresije i prirodnog tijeka bolesti, od nositelja odobrenja za stavljanje lijeka u promet traži se da podnese rezultate ispitivanja temeljene na odgovarajućem izvoru podataka uzetih iz registra bolesnika s alfa-manozidozom.
Linearity/(Non)linearity
Linearnost/nelinearnost
Concomitant long-term treatment with high doses of ibuprofen was noted as a potentially causative contributor to the occurrence of the event.
Zabilježeno je da je istodobno dugotrajno liječenje visokim dozama ibuprofena moguće doprinijelo nastanku tog događaja.
For infusion rate see section “Method of administration”.
Za brzinu infuzije vidjeti dio „Način primjene“.
Lamzede contains the active substance velmanase alfa which belongs to a group of medicines known as enzyme replacement therapies.
Lamzede sadrži djelatnu tvar velmanazu alfa, koja pripada skupini lijekova pod nazivom enzimska nadomjesna terapija.
Lamzede is administered through intravenous infusion.
Lamzede se primjenjuje putem intravenske infuzije.
Consequently hepatic and renal impairment are not expected to affect the pharmacokinetic of velmanase alfa.
Posljedično tome, ne očekuje se da će oštećenje funkcije jetre i bubrega utjecati na farmakokinetiku velmanaze alfa.
There are no clinical data on the effects of velmanase alfa on fertility.
Nema kliničkih podataka o učincima velmanaze alfa na plodnost.
To further evaluate the relationship, in instances of development of severe IRRs or lack or loss of treatment effect, patients should be tested for the presence of anti-velmanase alfa antibodies.
Radi daljnje procjene tog odnosa, u slučajevima razvoja teških reakcija povezanih s infuzijom ili izostanka ili gubitka terapijskog učinka, bolesnike je potrebno testirati na prisutnost protutijela na velmanazu alfa.
Lamzede should be reconstituted and administrated by a healthcare professional.
Lamzede mora rekonstituirati i primijeniti zdravstveni radnik.
A post-hoc multiparametric responders analysis supports the benefit of longer treatment with velmanase alfa in 87.9% of responders in at least 2 domains at last observation (table 4).
Post-hoc višeparametarska analiza bolesnika s terapijskim odgovorom govori u prilog koristi duljeg liječenja velmanazom alfa u 87,9% bolesnika s terapijskim odgovorom u najmanje 2 domene prilikom zadnjeg opažanja (tablica 4).
Lamzede is a white to off-white powder for solution for infusion, supplied in a glass vial.
Lamzede je bijeli do gotovo bijeli prašak za otopinu za infuziju, koji se isporučuje u staklenoj bočici.
The maximum dose of velmanase alfa in clinical studies was a single administration of 100 units/kg (approximately corresponding to 3.2 mg/kg).
Maksimalna doza velmanaze alfa u kliničkim ispitivanjima iznosila je jednokratno primijenjenih 100 jedinica/kg (što približno odgovara 3,2 mg/kg).
Antibodies may play a role in treatment-related reactions observed with the use of velmanase alfa.
Protutijela mogu igrati ulogu u reakcijama povezanima s liječenjem opaženima uz primjenu velmanaze alfa.
3MSCT (steps/min)
3MSCT (stepenica/minuti)
A total of 33 patients (20 males and 13 females, ranging in age from 6 to 35 years) were exposed to velmanase alfa in five clinical studies.
Ukupno su 33 bolesnika (20 muških i 13 ženskih, u dobi od 6 do 35 godina) bila izložena velmanazi alfa u pet kliničkih ispitivanja.
This means that due to the rarity of the disease, it has not been possible to obtain complete information on this medicinal product.
To znači da s obzirom na malu učestalost bolesti nije bilo moguće doći do potpunih informacija o ovom lijeku.
- The other ingredients are: disodium phosphate dihydrate, sodium dihydrogen phosphate dihydrate, mannitol and glycine.
- Drugi sastojci su: natrijev hidrogenfosfat dihidrat, natrijev dihidrogenfosfat dihidrat, manitol i glicin.
There is no experience with overdose of velmanase alfa.
Nema iskustva s predoziranjem velmanazom alfa.
Forcefully ejecting the water for injections from the syringe onto the powder should be avoided to minimise foaming.
Potrebno je izbjegavati snažno ubrizgavanje vode za injekcije iz štrcaljke u prašak kako bi se smanjilo stvaranje pjene.
If not used immediately, the reconstituted solution may be stored up to 24 hours at 2°C to 8°C.
Ako se ne primijeni odmah, rekonstituiranu otopinu može se čuvati do 24 sata na temperaturi od 2°C do 8°C.
In case the patient's condition deteriorates during ERT, cessation of treatment should be considered.
U slučaju pogoršanja stanja bolesnika tijekom enzimske nadomjesne terapije, potrebno je razmotriti prestanak liječenja.
Paediatric Study rhLAMAN-08.
Pedijatrijsko ispitivanje rhLAMAN-08.
Use of velmanase alfa in the age group 6 to 17 years is supported by evidence from clinical studies in paediatric (19 out of 33 patients) and adult patients.
Primjena velmanaze alfa u dobnoj skupini od 6 do 17 godina potkrijepljena je dokazima iz kliničkih ispitivanja u pedijatrijskih (19 od 33 bolesnika) i odraslih bolesnika.
Functional Domain Response Combined
Odgovor u funkcionalnoj domeni
The long-term efficacy and safety of velmanase alfa was investigated in the uncontrolled, open label, phase 3 clinical study rhLAMAN-10 in 33 subjects (19 paediatrics and 14 adults, from 6 to 35 years at treatment initiation) who previously participated in velmanase alfa studies.
Dugoročna djelotvornost i sigurnost velmanaze alfa ispitana je mjera u ispitivanju rhLAMAN-10, nekontroliranom, otvorenom kliničkom ispitivanju faze 3, u 33 ispitanika (19 pedijatrijskih i 14 odraslih, u dobi od 6 do 35 godina na početku liječenja) koji su prethodno sudjelovali u ispitivanjima velmanaze alfa.
Store and transport refrigerated.
POSEBNE MJERE ČUVANJA Čuvati i prevoziti na hladnom.
Hyperhidrosis(1)
hiperhidroza(1)
For the management of adverse reactions, see sections 4.4 and 4.8.
Za zbrinjavanje nuspojava, vidjeti dijelove 4.4 i 4.8.
*Velmanase alfa is produced in mammalian Chinese Hamster Ovary (CHO) cells using recombinant DNA technology.
*Velmanaza alfa proizvodi se u stanicama jajnika kineskog hrčka pomoću tehnologije rekombinantne
Overall effects of treatment were evaluated in the domains of pharmacodynamics (reduction of serum oligosaccharides), functional (three-minute stair climbing test (3MSCT), six-minute walking test (6MWT), and forced vital capacity (FVC) % predicted) and quality of life (childhood health assessment questionnaire (CHAQ) disability index (DI) and CHAQ VAS pain (visual analogue scale)).
Ukupni učinci liječenja bili su procijenjeni u domenama farmakodinamike (sniženje oligosaharida u serumu), funkcionalnosti (3-minutni test uspinjanja stepenicama (engl. three-minute stair climbing test, 3MSCT), šestominutni test hodanja (engl. six-minute walking test, 6MWT) i postotak predviđenog forsiranog vitalnog kapaciteta (FVC)) i kvalitete života (indeks onesposobljenosti (engl. disability index, DI) i vizualno-analogna ljestvica za bol (engl. visual analogue scale, VAS) na Upitniku za procjenu zdravlja djece (engl. childhood health assessment questionnaire, CHAQ)).
No dose adjustment is necessary for patients with renal or hepatic impairment.
Nije potrebna prilagodba doze u bolesnika s oštećenjem funkcije bubrega ili jetre.
Lamzede requires reconstitution and is intended for intravenous infusion only.
Lamzede se mora rekonstituirati i namijenjen je samo za primjenu intravenskom infuzijom.
Each vial is reconstituted by slowly injecting 5 mL of water for injections to the inside of the wall of each vial.
Sadržaj svake bočice rekonstituira se polaganim ubrizgavanjem 5 ml vode za injekcije uz unutarnju stijenku svake bočice.
Table 1: Adverse reactions reported in clinical studies in patients with alpha-mannosidosis treated with velmanase alfa
Tablica 1: Nuspojave zabilježene u kliničkim ispitivanjima u bolesnika s alfa-manozidozom liječenih velmanazom alfa
The most common adverse reactions observed were weight increase (18%), IRRs (9%), diarrhoea (12%), headache (9%), arthralgia (9%), increased appetite (6%) and pain in extremity (6%).
(9%), proljev (12%), glavobolja (9%), artralgija (9%), pojačan apetit (6%) i bol u ekstremitetu (6%).
Weight increase
- povećanje tjelesne težine
A volume of 10 mL sodium chloride solution should be administered through the infusion system to infuse the remaining fraction of Lamzede in the line to the patient.
Volumen od 10 ml otopine natrijevog klorida treba primijeniti kroz infuzijski sustav kako bi se bolesniku infundirao dio lijeka Lamzede koji je preostao u infuzijskom setu.
Aseptic technique is to be used during preparation.
Tijekom pripreme se mora primijeniti aseptični postupak.
After reconstitution, one mL of the solution contains 2 mg of velmanase alfa (10 mg / 5 mL).
Nakon rekonstitucije, jedan ml otopine sadrži 2 mg velmanaze alfa (10 mg / 5 ml).
Patient's weight (44 kg) × dose (1 mg/kg) = Patient dose (44 mg) 44 mg divided by 10 mg/vial = 4.4 vials, therefore, 5 vials should be reconstituted.
Tjelesna težina bolesnika (44 kg) × doza (1 mg/kg) = doza za bolesnika (44 mg) 44 mg podijeljeno s 10 mg/bočica = 4,4 bočice; stoga je potrebno rekonstituirati sadržaj
Velmanase alfa is a protein and is predicted to be metabolically degraded into amino acids.
Velmanaza alfa je protein te se predviđa da se metabolički razgrađuje na aminokiseline.
d) The reconstituted solution is to be slowly withdrawn from each vial with caution to avoid foaming in the syringe.
d) Rekonstituiranu otopinu treba polako i oprezno izvući iz svake bočice kako bi se izbjeglo stvaranje pjene u štrcaljki.
At steady-state after weekly infusion administration of 1 mg/kg of velmanase alfa, the mean maximum plasma concentration was about 8 µg/mL and was reached at 1.8 hours after the start of administration corresponding to the mean infusion duration time.
U stanju dinamičke ravnoteže nakon primjene infuzije velmanaze alfa u dozi od 1 mg/kg svakog tjedna, srednja vrijednost najveće koncentracije u plazmi bila je oko 8 µg/ml i postignuta je 1,8 sati nakon početka primjene, što odgovara srednjoj vrijednosti trajanja infuzije.
The clearance of velmanase alfa from plasma (mean 6.7 mL/h/kg) is consistent with a rapid cellular uptake of velmanase alfa via mannose receptors.
Klirens velmanaze alfa iz plazme (srednja vrijednost 6,7 ml/sat/kg) sukladan je brzom preuzimanju velmanaze alfa u stanice preko manoznih receptora.
IRRs include hypersensitivity in 3 patients and anaphylactoid reaction in 1 patient.
Reakcije povezane s infuzijom uključivale su preosjetljivost u 3 bolesnika i anafilaktoidnu reakciju u 1 bolesnika.
Final Study report:
Završno izvješće ispitivanja: studeni 2020.
Lamzede can be used during breastfeeding.
Lamzede se može primjenjivati tijekom dojenja.
Immunogenicity
Imunogeničnost
Administration of velmanase alfa may result in an IRR, including anaphylactoid reaction (see section 4.8).
Kao posljedica primjene velmanaze alfa može nastati reakcija povezana s infuzijom, uključujući anafilaktoidnu reakciju (vidjeti dio 4.8).
b) The powder should be reconstituted in the vial by a slow drop-wise addition of the water for injections down the inside of the vial and not directly onto the lyophilised powder.
b) Prašak se rekonstituira u bočici tako da se voda za injekcije sporo dodaje, kap po kap, niz unutrašnju stijenku bočice, a ne izravno u liofilizirani prašak.
Disodium phosphate dihydrate Sodium dihydrogen phosphate dihydrate Mannitol
natrijev hidrogenfosfat dihidrat natrijev dihidrogenfosfat dihidrat manitol glicin
In the phase 3 pivotal multi-centre, double-blind, randomised, placebo-controlled, parallel group study rhLAMAN-05, the efficacy and safety of repeated administrations of velmanase alfa over 52 weeks at a dose of 1 mg/kg given weekly as intravenous infusion were investigated.
U ispitivanju rhLAMAN-05, pivotalnom, multicetričnom, dvostruko slijepom, randomiziranom, placebom kontroliranom ispitivanju faze 3 na usporednim skupinama, tijekom 52 tjedna ispitivane su djelotvornost i sigurnost ponavljane primjene velmanaze alfa u dozi od 1 mg/kg koja se davala jedanput tjedno kao intravenska infuzija.
The patient should be observed for IRRs for at least one hour after the infusion according to clinical conditions and the physician's judgment.
Bolesnika je potrebno promatrati zbog reakcija povezanih s infuzijom najmanje jedan sat nakon infuzije, sukladno kliničkom stanju bolesnika i procjeni liječnika.
As with other enzyme replacement therapies, velmanase alfa does not cross the blood-brain-barrier.
Velmanaza alfa, kao ni druge enzimske nadomjesne terapije, ne prelazi krvno-moždanu barijeru.
- The active substance is velmanase alfa.
- Djelatna tvar je velmanaza alfa.
Each vial is for single use only.
Jedna bočica je samo za jednokratnu primjenu.
10 mg velmanase alfa
10 mg velmanaze alfa
If you experience any side effect like these, please tell your doctor immediately.
Ako imate bilo koju nuspojavu poput ovih, odmah obavijestite svog liječnika.
Patients
Bolesnici
This medicine must not be used if the reconstituted solution contains opaque particles or is discoloured.
Ovaj lijek se ne smije upotrijebiti ako primijetite da rekonstituirana otopina sadrži neprozirne čestice ili je promijenila boju.
Based on this criteria mild to moderate patients, presenting heterogeneous severity with ability to perform endurance tests, large variability of clinical manifestations and age of onset were enrolled.
Na temelju ovih kriterija, uključeni su bili bolesnici s blagim do umjerenim oblikom bolesti, koji su pokazivali heterogenu težinu bolesti, ali su bili sposobni obaviti testove izdržljivosti i u kojih su se kliničke manifestacije i dob u kojoj je bolest nastupila jako razlikovali.
SPECIFIC OBLIGATION TO COMPLETE POSTAUTHORISATION MEASURES FOR THE MARKETING AUTHORISATION UNDER EXCEPTIONAL CIRCUMSTANCES
POSEBNE OBVEZE ZA ISPUNJAVANJE MJERA NAKON DAVANJA ODOBRENJA KOD ODOBRENJA ZA STAVLJANJE LIJEKA U PROMET U IZNIMNIM OKOLNOSTIMA
In case symptoms such as angioedema (tongue or throat swelling), upper airway obstruction or hypotension occur during or immediately after infusion, anaphylaxis or an anaphylactoid reaction should be suspected.
U slučaju da se tijekom ili neposredno nakon infuzije pojave simptomi kao što su angioedem (oticanje jezika ili grla), opstrukcija gornjih dišnih puteva ili hipotenzija, potrebno je posumnjati na anafilaksiju ili anafilaktoidnu reakciju.
Responders Rates rhLAMAN-05 study n=25
Stope odgovora Ispitivanje rhLAMAN-05 n=25
The typical manifestations of the disease include distinctive facial features, mental retardation, difficulty in controlling movements, difficulties in hearing and speaking, frequent infections, skeletal problems, muscle pain and weakness.
Obično se bolest očituje prepoznatljivim crtama lica, mentalnom retardacijom, otežanom kontrolom pokreta, poteškoćama sa sluhom i govorom, učestalim infekcijama, problemima s kostima, boli u mišićima i slabošću.
10 mL vial (Type I glass) with a bromobutyl rubber stopper, an aluminium seal and a polypropylene flip off cap.
Bočica od 10 ml (staklo tipa I) s čepom od bromobutilne gume, aluminijskim prstenom i polipropilenskim zatvaračem.