blank Quick help
blank Maintenance news

Scheduled maintenance

Regular maintenance outages:
between 05.00 and 05.15 hrs CET (Monday to Sunday).

Other outages
Availability

2022.02.11

More...
blank News flashes

News Flashes

New version of the European Patent Register – SPC proceedings information in the Unitary Patent Register.

2024-07-24

More...
blank Related links

Release notes
European Patent Register Assistant
European Patent Register Resource Book
European Patent Register – pocket guide
FAQ - European Patent Register
Federated Register (Flyer)
Global Dossier (Flyer)
Register events
Deep link coverage
Federated Register content
The Patent Information Tour
Fair use charter

Extract from the Register of European Patents

EP About this file: EP2721151

EP2721151 - ANTI-TNF-THERAPY FOR THE MUCOPOLYSACCHARIDOSES AND OTHER LYSOSOMAL DISORDERS [Right-click to bookmark this link]
Former [2014/17]ANTI-TNF- THERAPY FOR THE MUCOPOLYSACCHARIDOSES AND OTHER LYSOSOMAL DISORDERS
[2017/30]
StatusNo opposition filed within time limit
Status updated on  12.10.2018
Database last updated on 19.10.2024
FormerThe patent has been granted
Status updated on  03.11.2017
FormerGrant of patent is intended
Status updated on  29.06.2017
FormerExamination is in progress
Status updated on  16.11.2016
Most recent event   Tooltip17.07.2020Lapse of the patent in a contracting state
New state(s): AL, IS		published on 19.08.2020  [2020/34]
Applicant(s)For all designated states
Mount Sinai School Of Medicine
One Gustave L. Levy Place
New York, New York 10029 / US
[2014/17]
Inventor(s)01 / SCHUCHMAN, Edward, H.
300 Lakeshore Drive
Haworth, NJ 07641 / US
02 / SIMONARO, Calogera, M.
300 Lakeshore Drive
Haworth, NJ 07641 / US
03 / STRIKER, Gary, E.
201 E. 79th Street
New York, NY 10075 / US
04 / VLASSARA, Helen
201 E. 79th Street
New York, NY 10075 / US
 [2014/17]
Representative(s)Potter Clarkson
Chapel Quarter
Mount Street
Nottingham NG1 6HQ / GB
[N/P]
Former [2017/49]Potter Clarkson LLP
The Belgrave Centre
Talbot Street
Nottingham NG1 5GG / GB
Former [2014/17]Potter Clarkson LLP
The Belgrave Centre
Talbot Street
Nottingham, NG1 5GG / GB
Application number, filing date12803458.420.06.2012
[2017/49]
WO2012US43369
Priority number, dateUS201161498946P20.06.2011         Original published format: US 201161498946 P
US201161569452P12.12.2011         Original published format: US 201161569452 P
[2014/17]
Filing languageEN
Procedural languageEN
PublicationType: A1 Application with search report
No.:WO2012177778
Date:27.12.2012
Language:EN
[2012/52]
Type: A1 Application with search report 
No.:EP2721151
Date:23.04.2014
Language:EN
The application published by WIPO in one of the EPO official languages on 27.12.2012 takes the place of the publication of the European patent application.
[2014/17]
Type: B1 Patent specification 
No.:EP2721151
Date:06.12.2017
Language:EN
[2017/49]
Search report(s)International search report - published on:US27.12.2012
(Supplementary) European search report - dispatched on:EP20.03.2015
ClassificationIPC:C12N13/00, A61K38/46, A61K31/737, A61K45/06
[2015/01]
CPC:
A61K31/737 (EP,US); A61K38/465 (EP,US); A61K38/46 (EP,US);
A61K45/06 (EP,US); A61P19/00 (EP); A61P3/00 (EP);
A61P3/10 (EP); A61P43/00 (EP) (-)
C-Set:
A61K31/737, A61K2300/00 (US,EP);
A61K38/46, A61K2300/00 (EP,US)
Former IPC [2014/17]C12N13/00
Designated contracting statesAL,   AT,   BE,   BG,   CH,   CY,   CZ,   DE,   DK,   EE,   ES,   FI,   FR,   GB,   GR,   HR,   HU,   IE,   IS,   IT,   LI,   LT,   LU,   LV,   MC,   MK,   MT,   NL,   NO,   PL,   PT,   RO,   RS,   SE,   SI,   SK,   SM,   TR [2014/17]
Extension statesBANot yet paid
MENot yet paid
TitleGerman:ANTI-TNF-THERAPIE FÜR MUCOPOLYSACCHARIDOSEN UND ANDERE LYSOSOMALE STÖRUNGEN[2014/17]
English:ANTI-TNF-THERAPY FOR THE MUCOPOLYSACCHARIDOSES AND OTHER LYSOSOMAL DISORDERS[2017/30]
French:THÉRAPIE ANTI-TNF CONTRE LES MUCOPOLYSACCHARIDOSES ET D'AUTRES MALADIES LYSOSOMIALES[2014/17]
Former [2014/17]ANTI-TNF- THERAPY FOR THE MUCOPOLYSACCHARIDOSES AND OTHER LYSOSOMAL DISORDERS
Entry into regional phase14.01.2014National basic fee paid 
14.01.2014Search fee paid 
14.01.2014Designation fee(s) paid 
14.01.2014Examination fee paid 
Examination procedure14.01.2014Examination requested  [2014/17]
16.10.2015Amendment by applicant (claims and/or description)
08.02.2016Despatch of a communication from the examining division (Time limit: M06)
16.08.2016Reply to a communication from the examining division
07.11.2016Despatch of a communication from the examining division (Time limit: M02)
04.01.2017Reply to a communication from the examining division
29.06.2017Communication of intention to grant the patent
27.10.2017Fee for grant paid
27.10.2017Fee for publishing/printing paid
27.10.2017Receipt of the translation of the claim(s)
Divisional application(s)The date of the Examining Division's first communication in respect of the earliest application for which a communication has been issued is  08.02.2016
Opposition(s)07.09.2018No opposition filed within time limit [2018/46]
Fees paidRenewal fee
30.06.2014Renewal fee patent year 03
29.06.2015Renewal fee patent year 04
27.06.2016Renewal fee patent year 05
27.06.2017Renewal fee patent year 06
Opt-out from the exclusive  Tooltip
competence of the Unified
Patent Court		 See the Register of the Unified Patent Court for opt-out data
Responsibility for the accuracy, completeness or quality of the data displayed under the link provided lies entirely with the Unified Patent Court.
Lapses during opposition  TooltipHU20.06.2012
AL06.12.2017
AT06.12.2017
CY06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
MK06.12.2017
NL06.12.2017
PL06.12.2017
PT06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
TR06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IS06.04.2018
IE20.06.2018
LU20.06.2018
MT20.06.2018
BE30.06.2018
CH30.06.2018
LI30.06.2018
[2020/33]
Former [2020/31]HU20.06.2012
AT06.12.2017
CY06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
MK06.12.2017
NL06.12.2017
PL06.12.2017
PT06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
TR06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
MT20.06.2018
BE30.06.2018
CH30.06.2018
LI30.06.2018
Former [2020/27]HU20.06.2012
AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
PT06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
TR06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
MT20.06.2018
BE30.06.2018
CH30.06.2018
LI30.06.2018
Former [2020/16]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
TR06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
MT20.06.2018
BE30.06.2018
CH30.06.2018
LI30.06.2018
Former [2020/08]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
MT20.06.2018
BE30.06.2018
CH30.06.2018
LI30.06.2018
Former [2019/24]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
BE30.06.2018
CH30.06.2018
LI30.06.2018
Former [2019/21]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
CH30.06.2018
LI30.06.2018
Former [2019/19]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
IE20.06.2018
LU20.06.2018
Former [2019/17]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
LU20.06.2018
Former [2019/15]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
MC06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/52]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SI06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/51]AT06.12.2017
CZ06.12.2017
DK06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/40]AT06.12.2017
CZ06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
PL06.12.2017
RO06.12.2017
RS06.12.2017
SE06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/39]AT06.12.2017
CZ06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
IT06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
PL06.12.2017
RS06.12.2017
SE06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/38]AT06.12.2017
CZ06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
PL06.12.2017
RS06.12.2017
SE06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/37]CZ06.12.2017
EE06.12.2017
FI06.12.2017
HR06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
PL06.12.2017
RS06.12.2017
SE06.12.2017
SK06.12.2017
SM06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/34]FI06.12.2017
HR06.12.2017
LT06.12.2017
LV06.12.2017
NL06.12.2017
RS06.12.2017
SE06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/24]FI06.12.2017
HR06.12.2017
LT06.12.2017
LV06.12.2017
RS06.12.2017
SE06.12.2017
BG06.03.2018
NO06.03.2018
GR07.03.2018
Former [2018/23]FI06.12.2017
HR06.12.2017
LT06.12.2017
RS06.12.2017
SE06.12.2017
NO06.03.2018
GR07.03.2018
Former [2018/22]FI06.12.2017
LT06.12.2017
NO06.03.2018
Documents cited:Search[X]WO2007095688  (UNIV AUSTRALIAN [AU], et al) [X] 1,8,10-14 * paragraphs [0022] , [0111] , [0118] , [ 0119]; claim 37 *;
 [A]  - BEUTLER E, "GAUCHER DISEASE: NEW MOLECULAR APPROACHES TO DIAGNOSIS AND TREATMENT", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, US, (19920508), vol. 256, no. 5058, doi:10.1126/SCIENCE.1589760, ISSN 0036-8075, pages 794 - 799, XP000371643 [A] 1,8,10-14 * the whole document *

DOI:   http://dx.doi.org/10.1126/science.1589760
 [XP]  - EFRAT ELIYAHU ET AL, "Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI", PLOS ONE, (20110822), vol. 6, no. 8, doi:10.1371/journal.pone.0022447, page e22447, XP055152282 [XP] 1,8,10-14 * the whole document *

DOI:   http://dx.doi.org/10.1371/journal.pone.0022447
 [T]  - EDWARD H. SCHUCHMAN ET AL, "Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses", PLOS ONE, (20130124), vol. 8, no. 1, doi:10.1371/journal.pone.0054459, page e54459, XP055152276 [T] * the whole document *

DOI:   http://dx.doi.org/10.1371/journal.pone.0054459
International search[Y]US2008292618  (WEISBART RICHARD H [US]);
 [Y]US2010160253  (COOMBE DEIRDRE ROMA [AU], et al);
 [Y]US2011091442  (BOYD ROBERT [US], et al);
 [Y]  - BIELICKI ET AL., "Advantages of Using Same Species Enzyme for Replacement Therapy in a Feline Model of Mucopolysaccharidosis Type VI", THE JOUMAL OF BIOLOGICAL CHEMISTRY, (1999), vol. 274, no. 51, pages 36335 - 36343, XP002457231

DOI:   http://dx.doi.org/10.1074/jbc.274.51.36335
 [Y]  - SIMONARO ET AL., "Involvement of the Toll-like receptor 4 pathway and use of TNF-alpha antagonists for treatment of the mucopolysaccharidoses", PNAS, vol. 107, no. 1, (2010), pages 222 - 227, URL: http://ukpmc.ac.uk/articles/PMC2806747/pdf/pnas.200912937.pdf, XP055139623

DOI:   http://dx.doi.org/10.1073/pnas.0912937107
 [Y]  - BAZIAN, ULTRA ORPHAN DRUGS FOR LYSOSOMAL STORAGE DISORDERS, ONLINE REPORT, (2009), pages 1 - 70, URL: http://www.specialisedservices.nhs.uk/library/22/A_Guideline_Comparison_and_Survey_of International_Current_Practice_in Ultra_Orphan_Drugs_for_Lysosomal_Storage_Disorders.pdf, XP055143829
by applicantEP0486526
 US5180715
 EP0585705
 EP0663836
 US5643892
 US5656272
 US5668116
 US5672347
 WO9841240
 WO9941399
 WO9957296
 US2001034328
 US6448380
 US6451983
 US6498237
 US2005208090
 US2007009500
 US7446098
 US2009111771
 US7951545
 US2011142818
    - "The Mucopolysaccharidoses", NEUFELD ET AL., METABOLIC AND MOLECULAR BASIS OF INHERITED DISEASE, MCGRAW-HILL, (2001), pages 3421 - 3452
    - "Storage Diseases of the Reticuloendothelial System", KOLODNY ET AL., NATHAN AND OSKI'S HEMATOLOGY OF INFANCY AND CHILDHOOD, 5th ed., W. B. SAUNDERS CO., (1998), vol. 2, pages 1461 - 1507
    - CLARKE, LA, "The Mucopolysaccharidoses: A Success of Molecular Medicine", EXPERT REV. MOL. MED., (2008), vol. 10
    - WRAITH ET AL., "Mucopolysaccharidosis Type II (Hunter Syndrome): A Clinical Review and Recommendations For Treatment in the Era of Enzyme Replacement Therapy", EUR. J. PEDIATR, (2008), vol. 167, pages 267 - 77, XP019590385
    - COX-BRINKMAN ET AL., "Ultrastructural Analysis of Dermal Fibroblasts in Mucopolysaccharidosis Type I: Effects of Enzyme Replacement Therapy and Hematopoietic Cell Transplantation", ULTRASTRUCT. PATHOL., (2010), vol. 34, pages 126 - 32
    - COPPA ET AL., "Effect of 6 Years of Enzyme Replacement Therapy on Plasma and Urine Glycosaminoglycans in Attenuated MPS I Patients", GLYCOBIOLOGY, (2010), vol. 20, pages 1259 - 73
    - DECKER ET AL., "Enzyme Replacement Therapy for Mucopolysaccharidosis VI: Growth and Pubertal Development in Patients Treated With Recombinant Human N-Acetylgalactosamine 4-Sulfatase", J. PEDIATR. REHABIL. MED., (2010), vol. 3, pages 89 - 100
    - VALAYANNOPOULOS ET AL., "Mucopolysaccharidosis VI Orphanet", J. RARE DIS., (2010), vol. 12, page 5
    - MCGILL ET AL., "Enzyme Replacement Therapy for Mucopolysaccharidosis VI From 8 Weeks of Age-A Sibling Control Study", CLIN. GENET, (2010), vol. 77, pages 492 - 498
    - COTUGNO ET AL., "Different Serum Enzyme Levels are Required to Rescue the Various Systemic Features of the Mucopolysaccharidoses", HUM. GENE THER., (2010), vol. 21, pages 555 - 69
    - HERATI ET AL., "Radiographic Evaluation of Bones and Joints in Mucopolysaccharidosis I and VII Dogs After Neonatal Gene Therapy", MOL. GENET. METAB., (2008), vol. 95, doi:doi:10.1016/j.ymgme.2008.07.003, pages 142 - 51, XP025546365

DOI:   http://dx.doi.org/10.1016/j.ymgme.2008.07.003
    - OSBORN ET AL., "Minicircle DNA-Based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of a-L-Iduronidase in Mice With Mucopolysaccharidosis Type I", MOL. THER, (2011), vol. 19, doi:doi:10.1038/mt.2010.249, pages 450 - 60, XP055177520

DOI:   http://dx.doi.org/10.1038/mt.2010.249
    - SIMONARO ET AL., "Bone Marrow Transplantation in Newborn Rats With Mucopolysaccharidosis Type VI: Biochemical, Pathological, and Clinical Findings", TRANSPLANTATION, (1997), vol. 63, pages 1386 - 93
    - SIMONARO ET AL., "Articular Chondrocytes From Animals With a Dermatan Sulfate Storage Disease Undergo a High Rate of Apoptosis and Release Nitric Oxide and Inflammatory Cytokines: A Possible Mechanism Underlying Degenerative Joint Disease in the Mucopolysaccharidoses", LAB INVESTI, (2001), vol. 81, pages 1319 - 1328
    - SIMONARO ET AL., "Joint and Bone Disease in Mucopolysaccharidosis VI and VII: Identification of New Therapeutic Targets and Biomarkers Using Animal Models", PEDIATR. RES., (2005), vol. 57, doi:doi:10.1203/01.PDR.0000156510.96253.5A, pages 701 - 707, XP003014242

DOI:   http://dx.doi.org/10.1203/01.PDR.0000156510.96253.5A
    - SIMONARO ET AL., "Mechanism of Glycosaminoglycan-Mediated Bone & Joint Disease: Implications for the Mucopolysaccharidoses & Other Connective Tissue Diseases", AMER. J. PATH., (2008), vol. 172, pages 112 - 122
    - SIMONARO ET AL., "Involvement of the Toll-Like Receptor 4 Pathway and Use of TNF-Alpha Antagonists for Treatment of the Mucopolysaccharidoses", PROC. NATL. ACAD. SCI., (2010), vol. 107, doi:doi:10.1073/pnas.0912937107, pages 222 - 7, XP055139623

DOI:   http://dx.doi.org/10.1073/pnas.0912937107
    - WEAVER, "Efficacy and Safety of the Anti-TNF Biologic Agents", MOD. RHEUMATOL., (2004), vol. 14, pages 101 - 112
    - CAILLAUD ET AL., "Gene Therapy in Lysosomal Diseases", BIOMED. PHARMACOTHER, (2000), vol. 54, pages 505 - 12
    - SUZUKI, K., "Lysosomal Diseases", SUZUKI, K., GRAHAM, D. I., LANTOS, P. K., GREENFIELD'S NEUROPATHOLOGY, ARNOLD, (2002), pages 653 - 735
    - SHULL ET AL., "Enzyme Replacement in a Canine Model of Hurler Syndrome", PROC. NATL. ACAD. SCI. USA, (1994), vol. 91, doi:doi:10.1073/pnas.91.26.12937, pages 12937 - 12941, XP002125064

DOI:   http://dx.doi.org/10.1073/pnas.91.26.12937
    - KAKKIS ET AL., "Overexpression of the Human Lysosomal Enzyme a-L-Iduronidase in Chinese Hamster Ovary Cells", PROT. EXPRESS. PURIF., (1994), vol. 5, doi:doi:10.1006/prep.1994.1035, pages 225 - 232, XP024799902

DOI:   http://dx.doi.org/10.1006/prep.1994.1035
    - WEINREB ET AL., "Effectiveness of Enzyme Replacement Therapy in 1028 Patients With Type 1 Gaucher Disease After 2 to 5 Years of Treatment: A Report From the Gaucher Registry", AM. J. MED., (2002), vol. 113, doi:doi:10.1016/S0002-9343(02)01150-6, pages 112 - 119, XP055300690

DOI:   http://dx.doi.org/10.1016/S0002-9343(02)01150-6
    - SCHIFFMAN ET AL., "Enzyme Replacement Therapy in Fabry Disease: A Randomized Controlled Trial", JAMA, (2001), vol. 285, pages 2743 - 2749
    - STEWARD, C. G., "Bone Marrow Transplantation for Genetic Diseases", STEWARD, C. G., FAIRBAIRN, L. J., TESTA, N. G., BLOOD CELL BIOCHEMISTRY, KLEWER ACADEMIC/PLENUM PUBLISHERS, (1999), vol. 8, pages 13 - 56
    - HOOGERBRUGGE ET AL., "Allogeneic Bone Marrow Transplantation for Lysosomal Storage Diseases. The European Group for Bone Marrow Transplantation", LANCET, (1995), vol. 345, pages 1398 - 1402
    - FAIRBAIRN ET AL., "Long-Term In Vitro Correction of a-L-Iduronidase Deficiency (Hurler Syndrome) in Human Bone Marrow", PROC. NATL. ACAD. SCI. U.S.A., (1996), vol. 93, pages 2025 - 2030
    - MEDIN ET AL., "Correction in Trans for Fabry Disease: Expression, Secretion, and Uptake of a-Galactosidase A in Patient-Derived Cells Driven by a High-Titer Recombinant Retroviral Vector", PROC. NATL. ACAD. SCI. USA, (1996), vol. 93, doi:doi:10.1073/pnas.93.15.7917, pages 7917 - 7922, XP002052041

DOI:   http://dx.doi.org/10.1073/pnas.93.15.7917
    - PAULY ET AL., "Complete Correction of Acid a-Glucosidase Deficiency in Pompe Disease Fibroblasts in Vitro, and Lysosomally Targeted Expression in Neonatal Rat Cardiac and Skeletal Muscle", GENE THERAPY, (1998), vol. 5, doi:doi:10.1038/sj.gt.3300609, pages 473 - 480, XP000934031

DOI:   http://dx.doi.org/10.1038/sj.gt.3300609
    - ZARETSKY ET AL., "Retroviral Transfer of Acid a-Glucosidase cDNA to Enzyme-Deficient Myoblasts Results in Phenotypic Spread of the Genotypic Correction by Both Secretion and Fusion", HUMAN GENE THERAPY, (1997), vol. 8, pages 1555 - 1563
    - GUO ET AL., "Elevated Plasma Chitotriosidase Activity in Various Lysosomal Storage Disorders", J. INHERIT. METAB. DIS, (1995), vol. 18, pages 717 - 722, XP000609807
    - DEN TANDT ET AL., "Marked Increase of Methylumbelliferyl-tetra-N-acetylchitotetraoside Hydrolase Activity in Plasma From Gaucher Disease Patients", J. INHERIT. METAB. DIS, (1996), vol. 19, pages 344 - 350
    - DODELSON DE KREMER ET AL., "Plasma Cchitotriosidase Activity in Argentinian Patients With Gaucher Disease, Various Lysosomal Diseases and Other Inherited Metabolic Disorders", MEDICINA (BUENOS AIRES, (1997), vol. 57, pages 677 - 684
    - CZARTORYSKA ET AL., "Changes in Serum Chitotriosidase Activity With Cessation of Replacement Enzyme (Cerebrosidase) Administration in Gaucher Disease", CLIN. BIOCHEM., (2000), vol. 33, pages 147 - 149
    - CZARTORYSKA ET AL., "Serum Chitotriosidase Activity in Gaucher Patients on Enzyme Replacement Therapy (ERT", CLIN. BIOCHEM., (1998), vol. 31, pages 417 - 420
    - MISTRY ET AL., "A Practical Approach to Diagnosis and Management of Gaucher's Disease", BAILLIERES CLIN. HAEMATOL, (1997), vol. 10, pages 817 - 838
    - YOUNG ET AL., "Plasma Chitotriosidase Activity in Gaucher Disease Patients who Have Been Treated Either by Bone Marrow Transplantation or by Enzyme Replacement Therapy With Alglucerase", J. INHERIT. METAB. DIS, (1997), vol. 20, pages 595 - 602
    - HOLLAK ET AL., "Marked Elevation of Plasma Chitotriosidase Activity. A Novel Hallmark of Gaucher Disease", J. CLIN. INVEST, (1994), vol. 93, doi:doi:10.1172/JCI117084, pages 1288 - 1292, XP000609695

DOI:   http://dx.doi.org/10.1172/JCI117084
    - GRELL ET AL., "The Transmembrane Form of Tumor Necrosis Factor is the Prime Activating Ligand of the 80 kDa Tumor Necrosis Factor Receptor", CELL, (1995), vol. 83, pages 793 - 802
    - CORCORAN ET AL., "Characterization of Ligand Binding by the Human p55 Tumour-Necrosis-Factor Receptor. Involvement of Individual Cysteine-Rich Repeats", EUR. J. BIOCHEM, (1994), vol. 223, doi:doi:10.1111/j.1432-1033.1994.tb19059.x, pages 831 - 840, XP000942145

DOI:   http://dx.doi.org/10.1111/j.1432-1033.1994.tb19059.x
    - CORCORAN ET AL., "Characterization of Ligand Binding by the Human p55 Tumour-Necrosis-Factor Receptor. Involvement of Individual Cysteine-Rich Repeats", EUR. J. BIOCHEM., (1994), vol. 223, doi:doi:10.1111/j.1432-1033.1994.tb19059.x, pages 831 - 840, XP000942145

DOI:   http://dx.doi.org/10.1111/j.1432-1033.1994.tb19059.x
    - The Merck Index, 11th ed., MERCK & CO, INC., (1989), page 7093
    - YOSHIDA ET AL., "Arylsulfatase B-Deficient Mucopolysaccharidosis in Rats", J. CLIN. INVEST, (1993), vol. 91, pages 1099 - 1104
    - KUNIEDA ET AL., "Mucopolysaccharidosis Type VI in Rats: Isolation of CDNAs Encoding Arylsulfatase B, Chromosomal Localization of the Gene, and Identification of the Mutation", GENOMICS, (1995), vol. 29, pages 582 - 587
    - SIMONARO ET AL., "Mechanism of Glycosaminoglycan-Mediated Bone & Joint Disease: Implications for the Mucopolysaccharidoses & Other Connective Tissue Diseases", AMER. J. PATH, (2008), vol. 172, pages 112 - 122
    - SEMENZA ET AL., "Respiratory Complications of Mucopolysaccharide Storage Disorders", MEDICINE, (1988), vol. 67, pages 209 - 19
    - BECKER ET AL., "Accumulation of Ceramide in the Trachea and Intestine of Cystic Fibrosis Mice Causes Inflammation and Cell Death", BIOCHEM. BIOPHYS. RES. COMMUN., (2010), vol. 17, doi:doi:10.1016/j.bbrc.2010.11.038, pages 368 - 74, XP027561195

DOI:   http://dx.doi.org/10.1016/j.bbrc.2010.11.038
    - METCALF ET AL., "Mechanism of Shortened Bones in Mucopolysaccharidosis VII", MOL. GENET. METAB., (2009), vol. 97, doi:doi:10.1016/j.ymgme.2009.03.005, pages 202 - 211, XP026157766

DOI:   http://dx.doi.org/10.1016/j.ymgme.2009.03.005
    - DECKER ET AL., "Enzyme Replacement Therapy for Mucopolysaccharidosis VI: Growth and Pubertal Development in Patients Treated With Recombinant Human N-Acetylgalactosamine 4-Sulfatase", J. PEDIATR. REHABIL. MED, (2010), vol. 3, pages 89 - 100
    - MIEBACH, E, "Enzyme Replacement Therapy in Mucopolysaccharidosis Type I. Treatment of Mucopolysaccharidosis Type II (Hunter syndrome) with Idursulfase: The Relevance of Clinical Trial End Points", ACTA PAEDIATR, (2005), pages 58 - 60
    - AUCLAIR ET AL., "Long-Term Intra-Articular Administration of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Feline Mucopolysaccharidosis VI", MOL. GEN. METAB., (2007), vol. 91, doi:doi:10.1016/j.ymgme.2007.04.009, page 352, XP022432047

DOI:   http://dx.doi.org/10.1016/j.ymgme.2007.04.009
    - KLAASEN ET AL., "Body Mass Index and Clinical Response to Infliximab in Rheumatoid Arthritis", ARTHRITIS RHEUM, (2011), vol. 63, pages 359 - 64
    - RODGERS ET AL., "Etanercept, Infliximab and Adalimumab for the Treatment of Psoriatic Arthritis: A Systematic Review and Economic Evaluation", HEALTH TECHNOL. ASSESS., (2011), vol. 15, doi:doi:10.3310/hta15100, pages 1 - 329, XP055314647

DOI:   http://dx.doi.org/10.3310/hta15100
    - CONNOR, V, "Anti-TNF Therapies: A Comprehensive Analysis of Adverse Effects Associated With Immunosuppression", RHEUMATOL. INT., (2011), vol. 31, doi:doi:10.1007/s00296-009-1292-x, pages 327 - 37, XP019883707

DOI:   http://dx.doi.org/10.1007/s00296-009-1292-x
    - SHINHAR ET AL., "Airway Management in Mucopolysaccharide Storage Disorders", ARCH. OTOLARYNGOL. HEAD NECK SURG, (2004), vol. 130, pages 233 - 237
The EPO accepts no responsibility for the accuracy of data originating from other authorities; in particular, it does not guarantee that it is complete, up to date or fit for specific purposes.