777
www.neurologiepropraxi.cz34080400107651332012142633073553814284484965465656086586857327828098518919109269661005
/33848040010732
Neurol.3461804001075593139165209227243
praxi.3720804001074672114152170186
2025;26(4):302-3093922804001074487128172185229272295337358373417461502528568612656
/46238040010732
NEUROLOGIE47018040010757101158205267310370429450493
PRO5216804001074591153
PRAXI5391804001074591148201221
30956558020012657114173
PŘEHLEDOVÉ4549200012660124182256312369444520587645
ČLÁNKY5216200012660115187259327395
Genová383936001063769110151188225
terapie40803600106296297139174191223
(vybraných)431836001062361100136172214255292325366389
neurologických472336001063971112147188219258298316350386425458499
onemocnění523836001063879111166206242283315356374
LITERATURA34157001234978134189250318368438498567
1.3407400853648
Aartsma-Rus409740083417496120148206239261299336363
A,7927400834256
Ginjaar868740083446098113145178199
IB,1088740083155163
Bushby11727400833574100138177209
K.14017400833551
The14727400833372108
importance16007400831573112151173198231269301335
of19567400833757
genetic3408400833873112147169185217
diagnosis578840083385386125163202229244270
for868840083195779
Duchenne9678400834684117155190229267302
muscular12908400835795122154192208240261
dystrophy.1572840083387098121143180220259290300
Med18928400835384120
Genet.340940083417210813916050094008312
2016;53:145-151.5289400833468100131143178211222252283316338369399430439
2.34010400853549
Birnkrant3991040083354868104132153184220239
DJ,6481040083456575
Bushby7331040083357095130167197
K,93910400833548
Bann99610400833566102138
CM,114310400834093104
et125710400833354
al.13201040083324556
Diagnosis13861040083455888125161197222235259
and165510400833167104
management17691040083568612215319022234011400835790127147
of49711400833754
Duchenne56111400834582112149182218255288
muscular85911400835793118148184198228248
dystrophy.11171140083386893115135170208245274282
Lancet14091140083296297125153172
Neurology.1589114008342711061251581712052402671853114008312
2018.18751140083346697126138
3.34012400853651
Bönnemann41212400833574113151186244276314353
CG,7851240083408296
Belluscio9011240083367187102139166198213251
BA,11731240083357690
Braun12841240083355790128166
S,147012400833346
et153612400833457
al.16131240083324760
Dystrophin169412400834578105129150188227266281319
Immunity34013400831573130168206221244277
after6331340083325075109130
Gene77913400834479117152
Therapy94713400833271106128161200231
for11941340083185677
Duchenne’s128713400834583115153188227265299312337
Muscular164013400835492118150188204236257
Dystrophy.1912134008346783401440083264971108148186217227
N582144008343
Engl63714400833167103118
J767144008324
Med.80414400835283119922144008312
2023;388(24):2294-2296.95014400833468101135147182216252271305336354366401436470506529559594628664677
4.34015400853651
European4051540083336990128167203235273
Medicines69115400835490129144176191229264291
Agency99515400834180115153185218
(EMA)122715400831851106148167
(online)14081540083185493108123161196213
https://www.16351540083375983123149162189209260314367378
ema.europa.eu/en/documents/product-information/libmeldy-epar-product-information_en.pdf.34016400833390122135169206226263301334346380417443474511536571609641677734768806827854879916937973101210481080110311221138117511931230125113081340136113761413145114761492150615441602163616513401740083387092128168201222241281303340380417450474493509547566604625684716738753791830867903941954994103310531064
5.34018400853650
European4041840083336990128167203235273
Medicines69018400835590129144176191229264291
Agency99518400834179114152184218
(EMA)122618400831952107149168
(online)14071840083195593109124162197214
https://www.16351840083375983122149162188209260313367377
ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf.34019400833390121134167204224261299331344377414439470507532566604635672728762799820847871908929965100310401071109411131128116511831220124012971329135013641401143814631491152715421580161416513402040083268411613917521424726828832835038742646449652054055659461365067273076378580083887691394998710001040108010991111
6.34021400853754
Hoon42221400834586127167
Lee61721400833269106
B,75121400833650
Deng82921400834784125166
S,102321400833347
Chiriboga10982140083418198120137179219261295
CA,142121400834186102
et155121400833559
al.16382140083324963
Newborn172921400834683137179220243284
Screening3402240083336790125162202218257298
for6602240083195881
Spinal7632240083337490130163180
Muscular96522400835695123157196212246268
Atrophy12552240083426587126167207239
in151622400831555
New159322400834683136
York17512240083367194126
State:189922400833456903402340083225871
Clinical4352340083415874113130163198214
Outcomes67323400834989113145184244281309
From10072340083335493154
the11852340083226299
First13082340083334972100125
31457234008335
Years.151623400833669103125154168
Neurology.1709234008344761141351721882252632942000234008313
2022;99(14):e1527-e1537.34024400833468101136148184219238270301318330365394424458491509545575605638670677
7.34025400853645
International4062540083155375108130169201223238276315347363
Commission78925400834077136194209235262277315354
on116325400833876
the12602540083216095
Clinical13752540083405671109124156189204
Use160025400834470105
of172525400833857
Human180225400834583141173211
Germline34026400834479100159174189227262
Genome62226400834479117156214249
Editing.8912640083327186108124161200213
Heritable112426400834580101116139172211227262
Human140626400834481139172210
Genome163526400834480118156214249
Editing.190426400833371863402740083223775114127
Kniha.4822740083357489127160173
2020.67027400833569101136148
8.34028400853652
Kirschner4112840083355172100132170208243264
J,69528400832436
Butoianu7502840083367396133148180218256
N,102528400834558
Goemans110228400834483118176209247273
N,139428400834558
et147128400833457
al.15472840083324760
European16272840083326990127167202234272
ad-19192840083327193
-hoc3402940083215997130
consensus48329400833167105131166204230267293
statement7892940083264981103136194228266287
on108929400833775
gene117729400833873110145
replacement13352940083205493109141173206264298336357
therapy17052940083226094115148187218
for19362940083185576
spinal3403040083266581118151166
muscular52030400835795121153191206238259
atrophy.7933040083325375112152190221231
Eur10373040083316686
J1134304008324
Paediatr11693040083346798134149184205225
Neurol.140530400834373110130165180193
2020;28:38-43.160930400833468101135148183217229264299321358391403
9.34031400853649
Kirschner4033140083355172100132170208243264
J,68031400832436
Bernert7293140083367192131166187211
G,95331400834458
Butoianu10243140083357395132148180218255
N,129231400834558
et136331400833456
al.14323140083324760
Update:150531400834483122155176210223
European17413140083326990127167202234272
ad-hoc3403240083327193132170203
consensus55732400833168106133168206232269296
statement8663240083264982104138196231269290
on117032400833775
gene125932400833873111146
replacement14193240083205494110142174208265301339360
therapy17933240083216095116149188219
for3403340083185678
spinal4313340083266581119151167
muscular61133400835895122154192207239260
atrophy.8853340083325475112152190221231
Eur11303340083316686
J1228334008324
Paediatr12633340083336797133148184205225
Neurol.1499334008343741101301651801678334008312
2024;51:7378.1704334008334681011331451802112192532873403440083346881
10.3403540085367085
Kohn44335400833572111149
DB,6103540083458194
Sadelain72235400833265104139155187202240
M,98035400835568
Glorioso10663540083446098119135173200238
JC.13223540083246578
Occurrence141835400834780110148169191225263295329
of176535400833757
leukaemia1840354008314498711715134036400833492107139
following495364008319567287126177192230269
gene78136400833873111146
therapy9433640083226095116149188220
of117936400833757
X-linked1252364008339587489127157191231
SCID.14993640083337388134145
Nat16613640083437999
Rev17743640083346495
Cancer.1882364008338731093403740083295839937400832131
2003;3(7):477-88.44537400833469105138149184202237256268303335369388424459473
11.3403840085366678
Landfeldt43138400833165103142161195211250272
E,71638400833346
Thompson77538400833372110168207234272311
R,109938400833549
Sejersen11613840083326782117138166200239
T,141338400833241
et146738400833456
al.15363840083324760
Life16093840083314766100
expectancy17223840083346610514117319823026834039400833165
at42639400833253
birth5003940083395576101139
in66039400831453
Duchenne73339400834684117155190228267302
muscular105539400835896122155193208241262
dystrophy:13383940083387097120142180219258289303
a1661394008332
systematic17093940083265986109143201234256271303
review3404040083215588104139191
and55140400833271110
meta‑analysis.68240400835893116149171204243275291323350365392405
European11084040083316687122159190226263
Journal13884040083246097118155191206
of161140400833554
Epidemiology.1682404008331668211814820522025627130734041400833564402414008312
2020;35:643-653.43041400833468101135148183216227262298331353390424457469
12.3404240085376986
Lugowska44742400833270110149201229260294
A,76242400834256
Poninska84042400833573112128167194224259
J,112042400832537
Krajewski11784240083365993109144197224255272
P,147142400833641
et153342400833557
al.16124240083324861
Population169542400833573113153168201224240279318
carrier3404340083326688110127162184
rates5464340083215578112140
of70743400833959
pathogenic7874340083397396135175216252291307340
ARSA114843400834279114156
gene132543400833975114150
mutations:149743400835897121154177193232272299312
is183043400831542
metachromatic1894434008358942140740083225690129151189250283306322355
leukodystrophy2517740083155190121160201234262286308347388428460
underdiagnosed?29987400833878118154176216232265305345384412448489517
PLoS3536740083346499130
One.36847400834684116380074008312
2011;6(6):e20218.21408400833468100128136172191224242254288323357390421452466
13.2140940085366883
Mendell22439400835490128167202217232
JR,2495940083246074
Al-Zaidy2589940083415779115148163202234
SA,2843940083327387
Lehman29509400833166104162194232
KJ,3202940083356375
et32979400833456
al.3373940083324760
Five-Year3453940083324780114137170201233254
Extension3727940083326521401040083225593120135173211
Results23681040083367198135150172200
of258510400833857
the26591040083226095
Phase277110400833673105132167
12955104008334
START300710400833266103139173
Trial3197104008333506698113
of332810400833756
Onasemnogene340110400834786118145180238276314354389214011400833873
Abeparvovec223411400834181117156189211246284316351384
in263811400831553
Spinal27121140083337287126158174
Muscular290611400835593120153191206239260
Atrophy.31871140083416284122162200231241
JAMA344911400832461115154
Neurol.3620114008343731101301661813800114008312
2021;78(7):834-841.214012400833468101133141174207226261280292327361396419455492522531
14.21401340085366883
Nařízení22361340083447697113141175213228
Evropského24761340083336486123162189218252290328
parlamentu28161340083397292108140197232270291329
a3157134008332
rady32041340083215392124
(ES)33401340083195185104
č.345613400833144
1394/2007.35131340083346497132157191225261293299
Úřední21401440083446599138176191
věstník23461440083326693116154169201
Evropské25621440083336587124163190220254
unie.28311440083387691125138
2007.298414400833468104137143
15.21401540085366883
Palfi22391540083356782100117
S,237215400833245
Gurruchaga243315400834482103125163195233265304337
JM,27851540083257993
Ralph28941540083356883122161
GS,30711540083437689
et317615400833456
al.32481540083324760
Long-term332415400833169107146168191225246304
safety36441540083265978113135169
and214016400833270110
tolerability22701640083225975110132165205220236251273307
of259816400833757
ProSavin,26761640083355693125159190206245257
a2954164008332
lentiviral30021640083155088110125158174195229244
vector-based32671640083326699123161182202243276303338378
gene366516400833974112147
therapy21401740083226298120155195228
for23901740083195880
Parkinson's24921740083366991123140180207247287299326
disease:28351740083395582118151179216229
a3085174008333
dose313517400833978106143
escalation,32991740083356397131147181203220259299312
open-363317400833879116156179
-label,21401840083213769109144160172
phase232718400833978110136171
1/225141840083345691
trial.2620184008322435991106119
The27541840083316898
Lancet.286518400832964101130159181193
2014;383(9923):1138-1146.307118400833468100131143178212246264297332365400417429459487517551574604632663699713
16.21401940085366984
Philippidis22371940083357287102116155194209248263288
A.253819400834155
Novartis260619400834482114146167191206232
Confirms285119400833976114131148169227253
Deaths311619400834580112133171197
of332519400833756
23394194008334
Patients34411940083356687102137174196223
Treated3677194008332498311421402040083225595
with22532040083516689127
Gene239820400834479117152
Therapy256920400833271106128161200231
Zolgensma.28182040083387591130165203229287319332
Genetic316920400834379117152174189221
Engineering340920400833270109124162197232254269307346
&3773204008340
Biotechnology21402140083355189111144177215253291307345385417
News257221400834580131158
August274521400834178117155181204
12,29652140083346476
2022.305721400833468101135148
17.21402240085366876
Rare22352240083356889123
Disease23772240083456087122153180215
Database.26102240083457899132171204231266278
X-linked2906224008339587489127157191231
adrenoleukodystrophy.31552240083327192126164203219253291321359398430457480502539579617648658
National21402340083447698113150188220235
Organization238823400834768106138175190219252273288326364
for27642340083185677
Rare28542340083356788122
Diseases.29882340083456086121152178213239253
https://rarediseases.32532340083375983122148160187207226259280313352367393427459485520546559
org/rare-diseases/adrenoleukodystrophy/.2140244008338599812414417719923325729731333937540743446949652255359261464868772574177781584588392395598310061028106511051144117511991205
Published336524400833673113129144171209244284
2019.367024400833469101131142
Accessed214025400834172103137164190225265
October242025400834780104141180216237
26,26732540083346780
2022.276825400833569101136149
18.21402640085377390
Rosenberg225326400833677106143183225263286326
SA,26022640083347793
Aebersold271826400834279120158181210251268310
P,305126400833642
Cornetta311626400834180104145182206232267
K,340726400833552
et348226400833559
al.35642640083335065
Gene365226400834582123160
Transfer21402740083335185124151172208230
into23922740083155376114
Humans252827400834483142175214242
–2792274008335
Immunotherapy284927400831574133172211250273312348370404444477
of334827400833757
Patients34272740083366991107143182205233
with36822740083516891130
Advanced214028400834281115150189223258299
Melanoma,246128400835692109142182222282315330
Using28132840083447187127167
Tumor-Infiltrating300328400833367127167190211228268287304320344366401424440479520
Lymphocytes35452840083326212216320324321402940083316692126154
Modified231629400835594135151169186222263
by260129400833871
Retroviral26942940083357194117155188204226260277
Gene299329400834480119155
Transduction.31702940083335185124151191230263288304343382396
N3589294008343
Engl365029400833168105120
J3789294008324
Med.2140304008353841212261304008312
323:570-578.229030400833467103115151185219243278312346360
19.21403140085366882
Sehara223531400833267105137159191
Y,243931400833543
Fujimoto2495314008333698499157195217253
KI,27613140083355164
Ikeguchi28383140083144478118155187225240
K.309131400833550
Persistent31543140083356889116131157180214251273
Expression344031400833265104126159186213227265303
of375631400833757
Dopamine-Synthesizing214032400834583123155213228265300323354384422443481516543557586602639678
Enzymes28313240083327099132191225252
15309632400833463
Years31723240083356697118145
After33303240083416084118139
Gene348232400834378116151
Transfer3645324008333508212014621403340083185374
in222733400831452
a2292334008332
Primate23393340083355671129161183216
Model256833400835593132167183
of276433400833756
Parkinson's28333340083356788118135172199237275286312
Disease.31603340083466187122154180215227
Hum340033400834278134
Gene354533400834171108138
Ther36943340083306898118
Clin21403440083385267103
Dev.2256344008344751052358344008312
2017;28(2):74-79.238634400833468100129139174207226260278290323354377408442453
20.21403540085367287
Shaimardanova224235400833270103118175208229267299337376408441
AA,26983540083418498
Chulpanova281135400834078115131170203241279311344
DS,31703540083457891
Solovyeva32763540083327187125157191225257290
VV,35813540083367685
et368135400833456
al.37523540083324760
Metachromatic214036400835490112145177215236273331364386401433
Leukodystrophy:259336400833166103133171210242269293314351391429460473
Diagnosis,30873640083456193132170208235249276289
Modeling,339636400835493132167183198236275287
and370436400833169108
Treatment21403740083335084116137196231269290
Approaches.244637400834180119141178211243281316343357
Front.28183740083325289128149163
Med.,299637400835590129142155
2020.316637400833469101135148
21.21403840085367082
Shaimardanova224038400833270103118175208229267299337376408441
AA,26983840083418498
Chulpanova281438400834078115130169202240279311344
DS,31753840083457891
Mulagovova328438400835492107139178217249286318351
AL,36533840083417286
et375738400833456
al.21403940083324760
Gene221539400834379117152
and238239400833169108
Cell25053940083407489104
Therapy262439400833372107128161200232
for28703940083195678
Epilepsy:29623940083337287103138177203236250
A3226394008341
Mini328339400835469107122
Review.342039400833570103118153204215
Front.36503940083325289127149162
Mol.214040400835493109122
Neurosci.,227740400834580117139176203235250263276
11256840400833462
May264540400835587118
2022.277840400833468101136149
22.21404140085367288
Stolberg,22454140083325491106145181202240253
Sheryl251541400833370105127161177
Gay270941400834376107
(November283341400831863102134168226265300322
28,31724140083346780
1999).326941400833565100135153164
"The34504140083215695130
Biotech35974140083365189111144177215
Death214042400834581113134172
of232842400833756
Jesse24004240083245986112147
Gelsinger".25634240083437994120135173212247268290300
The287842400833372107
New300042400834580131
York31474240083356990121
Times328342400833350107143169
Magazine.346842400835487126158188203241276288
23.21404340085367287
Turner22414340083336687126161182
TJ,24374340083325366
Zourray251643400833875113134156189220
C,275043400833952
Schorge281643400833365103141163201236
S,306643400833245
et312543400833456
al.31954340083314760
Recent326943400833570103136174196
advances347943400833171103136174206240267
in376043400831452
gene39407400833873111146
therapy4107740083226095116149189220
for4347740083195778
neurodevelopmental44467400833772110132169209244276310326364404462497536557590606
disorders5072740083385380118140178213234261
with5354740083506689127
epilepsy.55017400833474893940840083155089116148158
J411484008324
Neurochem.41518400834273109129164194231261317446784008312
2021;157(2):229-262.44958400833468101132140170200234250284302314349384418442472506539552
24.3940940085367186
US40409400834476
Food41299400833370109148
and42919400833270109
Drug44139400834667106145
Administration45719400834180137153190206232255276309331346384423
(online)5007940083195594109124162197214
https://www.5235940083375983123149162189209260314367378
fda.gov/vaccines-blood-biologics/skysona.3940104008318578910214117921123526730033236337841645147849953955559363267169473474978780384188189692895598110061036106910961135117312051219
25.39401140085367287
US404111400834476
Food413011400833270109148
and429211400833169108
Drug441411400834567105144
Administration457211400834179137152190205231254276309330346384422
(online)50081140083185493108123161196213
https://www.52351140083375983123150162189209260314367378
fda.gov/news-events/press-announcements/fda-approves-394012400831857891021411792102342703053563834034404725065445655936196566777117387647858178558939319691007103910731130116512031225125212781297133513671390142214611500152215591591162516511672
-first-gene-therapy-children-metachromatic-leukodystrophy.3940134008320385475101123142181215252286308330367401421453491522543575612626641679699732769791848882903935966100310231059111611471168118312141232124712811318134713831422145214791501152115581596163316631672
26.39401440085356983
US403414400834373
Food411814400833167104141
and427014400833167105
Drug438514400834565101138
Administration453414400834077132146182196220242262293313327363400
(online)49441440083195390104117153186202
https://www.fda.51571440083375779117142153178197246298349358376413444456
gov/vaccines-blood-biologics/tissue-tissue-products/elevidys.39401540083377410412615618721824726029633035537441242746350053755859661064666169773574877880482984986388891394898110031023103710621087112211551176121512351270130813431374139614221446147614901523155415681605163516601673
27.39401640085367180
Verhaart40341640083366890128160193214238
ECI,42871640083327388100
Robertson440216400833573113149170194222260299
A,471516400834155
Wilson47851640083577388114153191
IJ,49901640083154052
et505716400833456
al.51271640083324760
Prevalence,52021640083355590122155170205243275309321
incidence55371640083155339401740083314786121159191225
and418617400833270109
carrier43161740083316486107123158180
frequency45161740083194175115153188226258292
of482917400833756
5q–linked490617400833473109126141179209244283
spinal52101740083266581119152167
muscular53981740083579512215419239401840083154768
atrophy40231840083325475113152191221
–4260184008335
a4310184008332
literature4357184008315305286107140162200221255
review.46271840083215587103138189200
Orphanet484218400834566102139175211242263
Journal51171840083246096117154189204
of533418400833553
Rare54001840083346990119
Diseases.553218400834359394019400832454901141441684108194008312
2017;12:124.413619400833468100129139169199211241271303315
28.39402040085377491
Vission40522040083365481108124163203
research42762040083215684120153175207247
report(online)45442040083215697137159185205242282298313352389407
https://www.visionresearchreports.com/cell-and-gene-therapy-market/39387.497220400833961861271541681952172693243793904214384654805205595816163940214008326619311414518320423827831633836339040443647353155758662063565067370574378280484588091895397699810361071109311261165119612181277130913301361139514171445148015131546158016131619
29.39402240085367286
Vockley404722400833672105135152187219
J,428622400832437
Brunetti-Pierri43432240083365796134169191216231254291305340362384399
N,476322400834558
Chung484122400834078116154194
WK,505522400835894109
et518422400833457
al.52622240083314760
The534322400833272107
evolving54702240083356710512139402340083324785124
role40822340083215874109
of420823400833857
medical428323400835792132147179212227
geneticists452823400833873111146168183215230256280307
in485323400831553
the49232340083226095
era50362340083345588
of514223400833756
gene521623400833873111146
therapy:53802340083215994116149188219233
An394024400834179
urgency40322440083375897132170202235
to428024400832259
prepare.43522440083386094133166188222234
Genetics459924400834172108139160175204229
in483924400831551
Medicine.4901244008352831191341631782142455146244008312
2023;25:100022.517124400833468101136148183217228258290326362395429442
30.39402540085367287
Wassenberg404125400835789115142177215254290311349
T,440425400833241
Molero-Luis445925400835593109144165202225259296311337
M,481025400835468
Jeltsch4891254008325607598125158196
K,510025400833550
et516425400833456
al.52342540083324760
Consensus530825400834077115141176214241278304
guideline39402640083387691129164180195233267
for42202640083195678
the43112640083226095
diagnosis44192640083385385124162200226241267
and469926400833270109
treatment48212640083224377109130188223261283
of511726400833756
aromatic51862640083325390149181203218250
l-amino54492640083153769127142394027400833776
acid40282740083326479118
decarboxylase415927400833872105138159198237267301317349375410
(AADC)458227400831859102148189209
deficiency.48042740083387390107139154189227259292302
Orphanet511927400834768108146178216251273
Journal540527400832462100121160192207
of394028400833757
Rare40122840083356889123
Diseases41512840083456086121153180215242
(2017).44082840083195387118148167178
31.39402940085366981
Wadbudhe403429400835788127166204243281315
AM,436229400834196110
Meshram448429400835589116154175207265
RJ,47622940083356274
Tidke48482940083335088118152
SC.50132940083327386
Severe511129400833368100134155189
Combined531229400834077135174189227262301
Immunodeficiency394030400831572129167204242282316333350382397431469501534
(SCID)44873040083185090105150167
and466730400833269108
Its47883040083143764
New486530400834479129
Treatment50073040083335083115136194229266288
Modalities.530730400835593132164179194216231265292305
Cureus.394031400833872931221581824122314008312
2023415031400833468101135
Oct;15(10):430131400834779103117147177194226258276288
e47759.460431400833470103137170202213
32.39403240085367186
Wassenberg404132400835789115142177215254290311349
T,440432400833241
Molero-Luis445932400835593109144165202226259296311337
M,481032400835568
Jeltsch4892324008324597597125157195
K,510132400833549
et516432400833457
al.52353240083314760
Consensus530932400833976115141176214241278304
guideline39403340083387691129164180195233267
for42203340083195678
the43113340083226095
diagnosis44193340083385385124162200226241267
and469933400833270109
treatment48213340083224377109130188223261283
of511733400833756
aromatic51863340083325390149181203218250
l-amino54493340083153769127142394034400833876
acid40313440083316479118
decarboxylase416434400833872105138159199238268302318350376411
(AADC)459034400831960103149190211
deficiency.48163440083387390107139154189227259293303
Orphanet513434400834566102139175211242263
Journal54093440083246096117153189204
of394035400833553
Rare40063540083347090119
Diseases.413835400834459831131481722022274365354008312
2017;12:1229.439335400833468100129139169199211241271306340351
33.39403640085367187
Yin-Hsiu40473640083355290112159185200238
Ch,43053640083407991
Ni-Chung44173640083456083126164202240280
L,471736400833245
Sheng-Hong478336400833371106145184206253291330369
T,517336400833342
et523536400833457
al.53133640083314760
Efficacy5394364008333516886119152184218
and394037400833270109
safety40673740083265978112135168
of425337400833756
AAV2432737400834184118153
gene449837400833873111146
therapy46623740083215994116149188219
in489937400831452
children4969374008331698499138159194232
with52183740083516689127
aromatic53633740083325390148181202218250
L-amino39403840083314679137152190228
acid41823840083316379117
decarboxylase431338400833873106139160199238269302318351377412
deficiency:47393840083387390107139155189227259293306
an505938400833270
open-label,514338400833777112150173189221260296312324
phase548138400833877109394039400832661
1/240133940083345691
trial.4116394008322435990106118
Lancet424739400832964100130158179
Child44373940083377388102138
Adolesc458539400833873109124154178207
Health.4803394008342721081221431794981394008312
201750063940083346899128
Dec;1(4):265-273.514739400834580113126155170203220232267300334356386419453465
34.39404040085367388
Young404140400833569107145183
CM,423740400834095108
Quinn435840400834785100137175
C,454640400834053
Trusheim46114040083335088114152186201259
MR.488340400835490104
Durable500040400834583104137176192226
cell5239404008331658095
and534740400833269108
gene546840400833873110145
therapy39404140083226095116149188219
potential417641400833978100133171193208240256
patient44484140083397294109144182203
and466841400833270109
financial47944140083173471104142174189221236
impact:504741400831572112145177201214
US527841400834476
projections53704140083396198113148181205220394042400833776102
of405842400833757
product41314240083396097137175207231
approvals,437842400833271110132169201234250276289
patients46834240083387193108143181203231
treated,49304240083214377109130164204216
and516342400833169108
product52884240083386097137174206230
revenues.553542400832054394043400833266104142176203217
Drug417243400834465101137
Discov43214340083446083112147177
Today.451043400833163991351644672434008312
2022;27(1):17-30.470043400833468101135148183216232264278290320349368400435447
Máme28368200302208342553684
pro101968200302146245388
vás146068200302132264373
odborný28370800302141290439582681825951
kongres128770800302138277423567666797908
zcela28373400302117227357431564
zdarma.90073400302116265398497706839909
Právě28358200463196299471634812
jste11815820046383216334509
dokončili1776582004632124135727689701137120912811352
studium283620004631452634636697419371247
medicíny?1616620004633195037127849501022122213791496