Post on 09-Feb-2018
7/22/2019 Informationsbroschre fr rzte, Sphingotec
1/8
Grustlrcgsrfsflj cfob`dm crl`oot
spmfonjtcstprj-OY
7/22/2019 Informationsbroschre fr rzte, Sphingotec
2/8
Fo Icutsdmh`oi cotwfdlcht ecic dmtc Br`u fa H`ubc fmrcs Hcgcos Grustlrcgs, emrhfdm
wcrico ruoi >3.;;; Ocucrlr`oluonco if`nojstfzfcrt*. Ctw` cfo Rfcrtch `hhcr gctrjbbcoco
Br`uco fst gcf If`nojscstchhuon eoncr `hs 33 E`mrc uoi ecic zcmotc Br`u sjn`r eoncr `hs
93 E`mrc. Grustlrcgs fst i`aft ifc mubfnstc @rt icr Lrcgscrlr`oluon gcf Br`uco. Ec brm-
zcftfncr i`s Cotstcmco vjo Grustlrcgs crl`oot wfri, icstj mmcr sfoi ifc Dm`odco, fmo
crbjhnrcfdm zu gcm`oicho. Cotsdmcficoi fst gcr `udm, Wfsfljb`ltjrco br ifc Grustlrcgs-
cotwfdlhuon zu lcooco. B`ltjrco wfc z. G. @htcr, Cfotrftt icr crstco Wcnch gzw. icr Acoj-
p`usc, Ifdmtc ics Grustncwcgcs, gcr `udm ncoctfsdmc Rcroicruonco (Aut`tfjoco
sjnco`ootcr Grustlrcgsncoc) sfoi ofdmt gccfobhussg`r. @oicrc B`ltjrco, z. G. icr
Gjiy-A`ss-Foicx (GAF), wcrico iurdm Hcgcosncwjmomcftco wfc lrpcrhfdmc Gcttfnuon,
Cssncwjmomcftco uoi @hljmjhljosua gcstfaat. Aftmfhbc vjo capfrfsdmco Ajichhco
looco sjhdmc B`ltjrco zus`aaconcb`sst uoi foifvfiuchhc Wfsflco `om`oi gcl`ootcr
I`tco uoi Fobjra`tfjoco crrcdmoct wcrico. Aft [rjocurjtcosfo wuric ectzt cfo B`ltjr
ncbuoico, icr strlcr `hs `hhc gfsmcr gcl`ootco B`ltjrco aft ica Wfsflj, `o Grustlrcgs zu
crlr`olco, ljrrchfcrt.
Gcstcmt gcf cfocr Br`u cfo
crmmtcs Wfsflj, `o Grustlrcgs
zu crlr`olco, sj wfri fmr fo
ecica B`hh cfoc conc Rjrsjrnc,
z. G. iurdm Y`stuotcrsudmuonco
jicr rcnchafnc A`aaj-
nr`pmfc, capbjmhco.
*Lrcgs fo Icutsdmh`oi 7;;6/7;0;. 6. @usn`gc. Wjgcrt Ljdm-Fostftut (Mrsn) uoi ifc Ncschhsdm`bt
icr cpficafjhjnfsdmco Lrcgsrcnfstcr fo Icutsdmh`oi c.R. (Mrsn). Gcrhfo, 7;04
Wfsfljb`ltjrco br
Grustlrcgs sdmochhcrlcooco
7/22/2019 Informationsbroschre fr rzte, Sphingotec
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[rjocurjtcosfo 0-00> (prj-OY) mfoncnco fst cfo
st`gfhcs Br`nacot ics Ocurjtcosfo-Rjrhubcr-Ajhc-
lhs uoi wfri fo nhcfdmca Rcrmhtofs wfc i`s Ocu-
rjtcosfo ncgfhict. Cs stchht i`aft cfoco `udm fo
icr H`gjrrjutfoc cfosctzg`rco Turrjn`ta`rlcr br
Ocurjtcosfo i`r.
Ocurjtcosfo fst cfo [cptfi `us 04 @afojsurco
uoi wfri fa ^cscothfdmco fo ico O-Uchhco ics
Iooi`ras ncgfhict. Tcfoc Brcfsctzuon fo ifc Ufrlu-
h`tfjo iurdm ico Iooi`ra wfri iurdm tfcrfsdmc
Bcttc uoi Uudlcr a`ncghfdm gccfobhusst, cs wfrlt
u. `. `hs Tttfnuonsmjrajo. Icr OY0-Wczcptjr ics
Ocurjtcosfos wfri vjo a`hfnoco Grustlrcgszchhco
gcrcxprfafcrt. Ifc OY0-Wczcptjr-@ltfvfcruon iurdm
Ocurjtcosfo foiuzfcrt cfo uoljotrjhhfcrtcs, fov`sfvcsUchhw`dmstua iurdm Rcrmfoicruon icr @pjptjsc
vjo Grustlrcgszchhco. Ocurjtcosfo stfauhfcrt i`s
Grustlrcgszchhw`dmstua uoi wfri, u. `. br Grust-
lrcgszchhco, ifrclt `otf-`pjptjtfsdm wfrlcoi
gcsdmrfcgco. I` Ocurjtcosfo fo vfvj uoi fo vftrj
fost`gfh fst, fst scfoc ifrcltc Gcstfaauon br ifc
H`gjrrjutfoc ofdmt nccfnoct.
[rjocurjtcosfo 0-00> uoi Ocurjtcosfo
st`gfh Ocurjtcosfo(07=-09;)
fost`gfh
Ocurjacifo O
(070-073)
D-tcraCoic
Tfno`hpcptfi
(74 @3)
Lrpcrcfncocs
Mjrajo
Ocurjtcosfo
gcwfrlt
uoljotrjhhfcrtcs
Uchhw`dmstua
[rjocurjtcosfo (0-00>)
7/22/2019 Informationsbroschre fr rzte, Sphingotec
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Crncgofssc `us icr A`ha Ifct @oi D`odcr Ttuifc (AID)*fa E`mr 7;07 a`dmco
ico Uus`aacom`on zwfsdmco icr prj-OY-Ljozcotr`tfjo fa Ghut uoi ica Grust-
lrcgsrfsflj icuthfdm. Fo ifcscr prjspcltfvco Ljmjrtcostuifc wurico ruoi 7.;;;
Br`uco `us icr wcfghfdmco Ojra`hgcvhlcruon cfogczjnco, icoco zwfsdmco
0660 uoi 0669 Ghutph`sa`prjgco cotojaaco wurico. Ifcsc wurico `osdmhfc-
coi fo cfocr [rjgcog`ol nch`ncrt uoi 7;07 aft ica spmfonjtcst prj-OY ifc
ecwcfhfnco prj-OY-Ljozcotr`tfjoco crafttcht. Ifcsc ^crtc wurico aft ico gcr
ruoi 02 E`mrc vcrbjhntco uoi ncs`aachtco Fobjra`tfjoco zua Ncsuoimcfts-/
Crlr`oluonsst`tus icr fo ifc Ttuifc cfogczjncoco Br`uco vcrnhfdmco. 074 icr
Br`uco m`ttco fo icr Gcjg`dmtuonszcft Grustlrcgs cotwfdlcht. Mfcrgcf zcfntc sfdm,
i`ss crmmtc prj-OY-Ljozcotr`tfjoco aft cfoca lo`pp ircfb`dm crmmtco Wfsflj,
fa H`ubc icr bjhncoico 0;03 E`mrc `o Grustlrcgs zu crlr`olco, cfomcrncmco.
Ifc Crncgofssc icr AID-Ttuifc
ljootco fo cfocr wcftcrco Ttuifc,
icr A`ha [rcvcotfjo [rjecdtTtuifc (A[[, Rcrbbcothfdmuon fo
Rjrgcrcftuon), gcsttfnt uoi
mfosfdmthfdm icr Wfsflj`uss`nc sjn`r
ojdm gcrtrjbbco wcrico. @us
ico I`tco ifcscr Ttuifc crn`g sfdm
cfo d`. ocuob`dmcs Wfsflj zur Grust-
lrcgscotwfdlhuon gcf Rjrhfcnco
mjmcr prj-OY-Ljozcotr`tfjoco.
[rjocurjtcosfo5
icr Foifl`tjr brGrustlrcgsrfsflj
03 E`mrc
AID-Ttuifc A[[-Ttuifc
0; E`mrc 3 E`mrc 3 E`mrc
93
9;
43
4;
73
7;
03
0;
3
;
Grustlrcgscrlr`oluonco prj 0.;;; Br`uco
ofcirfncs prj-OY mjmcs prj-OY
Puchhco5 AID- uoi A[[-Ttuifc
* Ach`oicr, J, ct `h., [h`sa` [rjocurjtcosfo `oi Fodficodc jb If`gctcs, D`rifjv`sduh`r
Ifsc`sc, Grc`st D`odcr, `oi Ajrt`hfty. E@A@ (Ejuro`h jb tmc @acrfd`o Acifd`h @ssjdf`tfjo).
0;. Jdtjgcr 7;07, Gi. 4;=, 09, T. 0926->3.
7/22/2019 Informationsbroschre fr rzte, Sphingotec
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fo scfocr @uss`nclr`bt. Ifc crafttchtc prj-OY-
Ljozcotr`tfjo crh`ugt cfoc Cfostubuon ncncogcr
ica ojra`hco Grustlrcgsrfsflj cfocr Br`u.
**z. G. @htcr, Cfoo`mac ghutirudlscolcoicr Afttch,
Mjrajocrs`tztmcr`pfc, jr`hc Ljotr`zcptfv`,
Gfhiuonsofvc`u, @htcr gcf Aco`rdmc, @htcr gcf
0. Tdmw`oncrsdm`bt, @oz`mh icr Lfoicr, Acojp`usco-
Tt`tus, systjhfsdmcr Ghutirudl, GAF, If`gctcs achhftus,
W`udmvcrm`htco, vjrm`oicoc l`rifjv`sluhrc
Crlr`oluon, crghfdmc Rjrgch`stuon, Ljozcotr`tfjoco
MIH, HIH uoi Fosuhfo.
Aftmfhbc icr br ifcsc Br`uco crafttchtco prj-OY-
Ljozcotr`tfjoco ljootc i`s Ucmo-E`mrcs-Wfsflj br
ifc Cotwfdlhuon vjo Grustlrcgs rclh`ssfbfzfcrt
wcrico, sji`ss
72 % icr Br`uco aft sptcrcr Grustlrcgsgfhiuon fo
cfoc mmcrc uoi
70 % icr Br`uco jmoc sptcrc Grustlrcgsgfhiuon fo
cfoc ofcirfncrc Wfsfljl`tcnjrfc cfoncstubt wurico.
Ifc Wfsfljgcrcdmouon br ifc fo ifc AID-Ttuifc
cfoncsdmhjsscoco Br`uco crbjhntc uotcr Cfogczfcmuon
gcrcfts gcl`ootcr Wfsfljp`r`actcr**. Cfoc ifrcltc
Ljrrch`tfjo zwfsdmco prj-OY uoi GAF `hs cfoca
icr wfdmtfnco Wfsfljp`r`actcr crn`g sfdm `us ico
Ttuifcoi`tco ofdmt. Ifc Gcstfaauon ics prj-OY
hfcbcrt sjaft zustzhfdmc Fobjra`tfjoco zu gfsmcr
gcl`ootco Wfsfljb`ltjrco uoi gcrtrfbbt ifcsc
prj-OY5
wcofncr fst gcsscr
icuthfdm
vcrrfoncrtcs Wfsflj
2; 07; 0=; pajh/H prj-OY
vcrrfoncrtcs Wfsflj
crmmtcs Wfsflj
icuthfdm
crmmtcs Wfsflj
7/22/2019 Informationsbroschre fr rzte, Sphingotec
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Icr O`dmwcfs ics [cptfi-Gfja`rlcrs prj-OY nfgt `hhco Br`uco, uo`gmonfn vjo
`oicrco, `udm ncoctfsdm gcifontco B`ltjrco, @usluobt gcr cfo crmmtcs
Crlr`oluonsrfsflj. Aft icr Ljozcotr`tfjosacssuon vjo prj-OY wfri cfo jgecltfv
acssg`rcr Uust`oi fa Lrpcr crafttcht, icr zw`r zua Ucftpuolt icr Acssuon ojdm
lcfo ncsuoimcfthfdmcs [rjghca i`rstchhco auss, `gcr gcr cfoco honcrco Ucft-
r`ua ifc Cotwfdlhuon vjo Grustlrcgs gcnostfnco jicr `ozcfnco l`oo. I`s gcr
ifc prj-OY-Gcstfaauon i`rstchhg`rc Wfsflj fst `hsj lcfoc @uss`nc, jg cfoc Br`u
gcrcfts Grustlrcgs cotwfdlcht m`t jicr jg cfoc Br`u fa H`ubc fmrcs Hcgcos t`tsdm-
hfdm `o Grustlrcgs crlr`olco wfri. Cs vcrnhcfdmt ecijdm ifc Mubfnlcft `ubtrctco-
icr Grustlrcgscrlr`oluonco zwfsdmco Br`uco aft foftf`h ncrfonca Wfsflj (ncrfonc
prj-OY-Ljozcotr`tfjo) uoi Br`uco aft mjmca Wfsflj (mjmc prj-OY-Ljozcotr`tfjo).
Aftmfhbc ics spmfonjtcst prj-OY
hsst sfdm i`s lrpcrcfncoc Mjrajo
Ocurjtcosfo fa Ghut o`dmwcfsco.Ifc Ghutcoto`mac crbjhnt fo icr
@rztpr`xfs, ifc @o`hysc fo cfoca icr
Cfoscoich`gjrs, ifc aft spmfonjtcd
zus`aaco`rgcftco.
^chdmc Fobjra`tfjohfcbcrt icr prj-OY-^crt:
7/22/2019 Informationsbroschre fr rzte, Sphingotec
7/8
tfcrfsdmc Bcttc uoi Uudlcr, rcnuhfcrt wfri. Fosjbcro
stchht ifc nczfchtc Wciuzfcruon vjo tfcrfsdmco Bcttco fo
icr Cromruon cfoc mjbbouonsvjhhc [rvcotfjo i`r.
M`t ifc Gcstfaauon icr [rjocurjtcosfo-Ljozcotr`-
tfjo nczcfnt, i`ss cfo crmmtcs Grustlrcgsrfsflj
gcstcmt, capbfcmht sfdm fo ecica B`hh cfoc conc Rjr-
sjrnc, i` cfoc brmc Crlcoouon ifc Mcfhuonsdm`odco
fo icr Wcnch icuthfdm vcrgcsscrt.
Uwfsdmco icr Cromruon uoi `oicrco B`ltjrco ics
Hcgcosstfhs (z. G. lrpcrhfdmc @ltfvftt, Ncwfdmt) uoi
icr Cotwfdlhuon vjo Lrcgs, sj udm Grustlrcgs, gc-
stcmt cfo Uus`aacom`on. U`mhrcfdmc Ttuifco m`gco
i`s Ufch, zu cfocr wcftcrco Lhruon ics Uus`aaco-
m`ons zwfsdmco Cromruon uoi Lrcgs sjwfc oicrco
dmrjofsdmco Crlr`oluonco gcfzutr`nco uoi sjaft
nco`ucrc Capbcmhuonco br cfoc ncsuoimcftsbricro-
ic Cromruon zu cranhfdmco. Tpczfchh ifc Uubumrtfcrfsdmcr Bcttc stcmt mubfn fa Bjlus sjhdmcr Sotcr-
sudmuonco. Aft icr Gcstfaauon ics prj-OY wfri ifc
Anhfdmlcft crbboct, z. G. i`s `us ica Cromruons-
vcrm`htco rcsuhtfcrcoic Wfsflj jgecltfvfcrg`r zu a`-
dmco, i` ifc Brcfsctzuon vjo Ocurjtcosfo gcl`ootcr-
a`co gcr ifc O`mruons`ubo`mac, spczfchh iurdm
Ifc Gcstfaauon
ics prj-OY
jgecltfvfcrt
gccfobhussg`rc
Wfsfljb`ltjrco
7/22/2019 Informationsbroschre fr rzte, Sphingotec
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^cftcrc Fobjra`tfjoco sfoi gcf spmfonjtcd crmhthfdm.
spmfonjtcd NagM
Ocucoijrbstr`c 03@, 02>20 Mcoofnsijrb
Ychcbjo ; 44 ;7/7 ;3 23-;
fobjKspmfonjtcd.ic, www.spmfonjtcd.ic
[rjocurjtcosfo (prj-OY) fst icr cfozfnc prifltfvc [h`sa`-Gfja`rlcr, icr cfoc
@uss`nc gcr ifc Wfsfljvcroicruon zur Cotwfdlhuon vjo Grustlrcgs hfcbcrt. Ifcs
fst fo acmrcrco lhfofsdmco Ttuifco st`tfstfsdm sfnofbfl`ot i`rnchcnt wjrico. Aft
ica spmfonjtcst prj-OY hsst sfdm ifc [rjocurjtcosfo-Ljozcotr`tfjo zuvcrhssfn
uoi sdmochh gcstfaaco.
Ifc prj-OY-Gcstfaauon crbjhnt fa H`gjr us cfocr fo icr [r`o`hytfl uoi @o`hytflcfob`dm uoi st`gfh zu m`oim`gcoico, odmtcro cotojaacoco CIY@-Ghutprjgc
aftmfhbc cfocs h`gjrnonfnco faauoif`nojstfsdmco Rcrb`mrcos (Faauoj`ss`y).
Bjtjs5074rb,spmfonjtcd|^C-IC-[OYZW;;7