HRP20201827T1 - Postupak za predviđanje odgovora na liječenje bolesti farmakološkim šaperonom - Google Patents
Postupak za predviđanje odgovora na liječenje bolesti farmakološkim šaperonom Download PDFInfo
- Publication number
- HRP20201827T1 HRP20201827T1 HRP20201827TT HRP20201827T HRP20201827T1 HR P20201827 T1 HRP20201827 T1 HR P20201827T1 HR P20201827T T HRP20201827T T HR P20201827TT HR P20201827 T HRP20201827 T HR P20201827T HR P20201827 T1 HRP20201827 T1 HR P20201827T1
- Authority
- HR
- Croatia
- Prior art keywords
- deoxygalactonojirimycin
- pharmaceutically acceptable
- acceptable salt
- use according
- gal
- Prior art date
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- 108010006519 Molecular Chaperones Proteins 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 230000000144 pharmacologic effect Effects 0.000 title 1
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 claims 15
- 229950007469 migalastat Drugs 0.000 claims 15
- 150000003839 salts Chemical class 0.000 claims 15
- 101000718529 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Alpha-galactosidase Proteins 0.000 claims 13
- NNISLDGFPWIBDF-MPRBLYSKSA-N alpha-D-Gal-(1->3)-beta-D-Gal-(1->4)-D-GlcNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)C(O)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)O)[C@@H](O)[C@@H](CO)O1 NNISLDGFPWIBDF-MPRBLYSKSA-N 0.000 claims 13
- 230000035772 mutation Effects 0.000 claims 12
- 102220045124 rs587781846 Human genes 0.000 claims 6
- 102220103088 rs142193069 Human genes 0.000 claims 4
- 102220486135 Alpha-galactosidase A_C56F_mutation Human genes 0.000 claims 3
- 102220486134 Alpha-galactosidase A_C56Y_mutation Human genes 0.000 claims 3
- 102220486228 Alpha-galactosidase A_G35R_mutation Human genes 0.000 claims 3
- 102220486653 Alpha-galactosidase A_I242N_mutation Human genes 0.000 claims 3
- 102220486351 Alpha-galactosidase A_Q280H_mutation Human genes 0.000 claims 3
- 102220466993 Annexin A3_I219N_mutation Human genes 0.000 claims 3
- 102220581245 Cell cycle regulator of non-homologous end joining_S247C_mutation Human genes 0.000 claims 3
- 102220493496 EF-hand calcium-binding domain-containing protein 12_E66G_mutation Human genes 0.000 claims 3
- 102220632134 N-sulphoglucosamine sulphohydrolase_P293T_mutation Human genes 0.000 claims 3
- 102220471509 Sarcoma antigen 1_Y216D_mutation Human genes 0.000 claims 3
- 102220514548 Somatostatin receptor type 1_N34K_mutation Human genes 0.000 claims 3
- 102200076932 c.179C>T Human genes 0.000 claims 3
- 102200076455 c.254G>A Human genes 0.000 claims 3
- 102200077291 c.893A>G Human genes 0.000 claims 3
- 102200038481 rs118192205 Human genes 0.000 claims 3
- 102200023312 rs121912940 Human genes 0.000 claims 3
- 102200126544 rs121917836 Human genes 0.000 claims 3
- 102220104213 rs140506267 Human genes 0.000 claims 3
- 102220141945 rs140804035 Human genes 0.000 claims 3
- 102220260525 rs1554097567 Human genes 0.000 claims 3
- 102200129595 rs1556318633 Human genes 0.000 claims 3
- 102220025220 rs199476357 Human genes 0.000 claims 3
- 102220244117 rs200243989 Human genes 0.000 claims 3
- 102200076374 rs28935195 Human genes 0.000 claims 3
- 102220011918 rs386134171 Human genes 0.000 claims 3
- 102220096855 rs386833401 Human genes 0.000 claims 3
- 102200076869 rs398123201 Human genes 0.000 claims 3
- 102220056052 rs398123224 Human genes 0.000 claims 3
- 102200155811 rs56165452 Human genes 0.000 claims 3
- 102220057314 rs730880450 Human genes 0.000 claims 3
- 102220277657 rs746045584 Human genes 0.000 claims 3
- 102220098947 rs749510661 Human genes 0.000 claims 3
- 102220257452 rs763104308 Human genes 0.000 claims 3
- 102220097736 rs774586397 Human genes 0.000 claims 3
- 102200076503 rs782197638 Human genes 0.000 claims 3
- 102220075588 rs796053298 Human genes 0.000 claims 3
- 102200076871 rs797044613 Human genes 0.000 claims 3
- 102220082942 rs863223870 Human genes 0.000 claims 3
- 102200086712 rs879253779 Human genes 0.000 claims 3
- 102220198395 rs976164079 Human genes 0.000 claims 3
- 102220486668 Alpha-galactosidase A_D244H_mutation Human genes 0.000 claims 2
- 102220481480 Alpha-galactosidase A_E338K_mutation Human genes 0.000 claims 2
- 102220486406 Alpha-galactosidase A_M187V_mutation Human genes 0.000 claims 2
- 102220480371 Alpha-galactosidase A_P409T_mutation Human genes 0.000 claims 2
- 102220512848 Heterogeneous nuclear ribonucleoprotein A1-like 2_N215D_mutation Human genes 0.000 claims 2
- 102200077213 rs104894852 Human genes 0.000 claims 2
- 102220115711 rs1114167345 Human genes 0.000 claims 2
- 102200069129 rs121434329 Human genes 0.000 claims 2
- 102200128188 rs121908803 Human genes 0.000 claims 2
- 102200093291 rs1555211982 Human genes 0.000 claims 2
- 102200077342 rs190347120 Human genes 0.000 claims 2
- 102220007476 rs267606804 Human genes 0.000 claims 2
- 102200131211 rs267607647 Human genes 0.000 claims 2
- 102200077255 rs28935488 Human genes 0.000 claims 2
- 102220001248 rs28940895 Human genes 0.000 claims 2
- 102200005819 rs3218725 Human genes 0.000 claims 2
- 102200078669 rs370595480 Human genes 0.000 claims 2
- 102200013203 rs387906468 Human genes 0.000 claims 2
- 102220028749 rs398123217 Human genes 0.000 claims 2
- 102220028761 rs398123227 Human genes 0.000 claims 2
- 102200020122 rs6054 Human genes 0.000 claims 2
- 102220178599 rs61760967 Human genes 0.000 claims 2
- 102200077331 rs727505292 Human genes 0.000 claims 2
- 102220057309 rs730880441 Human genes 0.000 claims 2
- 102200065197 rs760277934 Human genes 0.000 claims 2
- 102220064075 rs762381137 Human genes 0.000 claims 2
- 102220480233 Adhesion G-protein coupled receptor D1_P293A_mutation Human genes 0.000 claims 1
- 102220486386 Alpha-galactosidase A_A288D_mutation Human genes 0.000 claims 1
- 102220486126 Alpha-galactosidase A_E48D_mutation Human genes 0.000 claims 1
- 102220486513 Alpha-galactosidase A_G163V_mutation Human genes 0.000 claims 1
- 102220486399 Alpha-galactosidase A_L300F_mutation Human genes 0.000 claims 1
- 102220486311 Alpha-galactosidase A_P265R_mutation Human genes 0.000 claims 1
- 102220486021 Alpha-galactosidase A_R112S_mutation Human genes 0.000 claims 1
- 102220486115 Alpha-galactosidase A_R49L_mutation Human genes 0.000 claims 1
- 102220486129 Alpha-galactosidase A_R49S_mutation Human genes 0.000 claims 1
- 102220486655 Alpha-galactosidase A_W236L_mutation Human genes 0.000 claims 1
- 102220486015 Alpha-galactosidase A_W95S_mutation Human genes 0.000 claims 1
- 102000017927 CHRM1 Human genes 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 102200076863 c.119C>T Human genes 0.000 claims 1
- 102220444978 c.41T>C Human genes 0.000 claims 1
- 102220362014 c.702C>G Human genes 0.000 claims 1
- 239000001354 calcium citrate Substances 0.000 claims 1
- 102220282937 rs1044486334 Human genes 0.000 claims 1
- 102200086359 rs104894617 Human genes 0.000 claims 1
- 102200076873 rs104894831 Human genes 0.000 claims 1
- 102200076926 rs104894836 Human genes 0.000 claims 1
- 102200076919 rs1057521047 Human genes 0.000 claims 1
- 102220023973 rs112534524 Human genes 0.000 claims 1
- 102200076472 rs113173389 Human genes 0.000 claims 1
- 102200125504 rs121908130 Human genes 0.000 claims 1
- 102200038969 rs137852289 Human genes 0.000 claims 1
- 102200145385 rs137853133 Human genes 0.000 claims 1
- 102220270545 rs1383831944 Human genes 0.000 claims 1
- 102200036676 rs143947056 Human genes 0.000 claims 1
- 102220152464 rs144140791 Human genes 0.000 claims 1
- 102220319050 rs144524958 Human genes 0.000 claims 1
- 102220312151 rs1555152518 Human genes 0.000 claims 1
- 102220335256 rs1555407424 Human genes 0.000 claims 1
- 102220252826 rs1555980053 Human genes 0.000 claims 1
- 102200077127 rs1555984869 Human genes 0.000 claims 1
- 102200076560 rs1555985175 Human genes 0.000 claims 1
- 102200094055 rs17576 Human genes 0.000 claims 1
- 102200002868 rs199474731 Human genes 0.000 claims 1
- 102220087827 rs28935489 Human genes 0.000 claims 1
- 102200077252 rs28935495 Human genes 0.000 claims 1
- 102220082624 rs370063423 Human genes 0.000 claims 1
- 102220071048 rs373261115 Human genes 0.000 claims 1
- 102200076921 rs398123205 Human genes 0.000 claims 1
- 102220028747 rs398123213 Human genes 0.000 claims 1
- 102200132780 rs587783841 Human genes 0.000 claims 1
- 102200077248 rs730880451 Human genes 0.000 claims 1
- 102200068463 rs760106433 Human genes 0.000 claims 1
- 102200021815 rs765219004 Human genes 0.000 claims 1
- 102220166398 rs766522793 Human genes 0.000 claims 1
- 102220059876 rs786201800 Human genes 0.000 claims 1
- 102220055601 rs797044497 Human genes 0.000 claims 1
- 102200139295 rs80338771 Human genes 0.000 claims 1
- 102200058458 rs80358201 Human genes 0.000 claims 1
- 102220085015 rs863225303 Human genes 0.000 claims 1
- 102220087834 rs869312324 Human genes 0.000 claims 1
- 102200160956 rs869320708 Human genes 0.000 claims 1
- 102220099779 rs878854567 Human genes 0.000 claims 1
- 102220123424 rs886043407 Human genes 0.000 claims 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
- G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
- G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/90—Enzymes; Proenzymes
- G01N2333/914—Hydrolases (3)
- G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
- G01N2333/94—Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/38—Pediatrics
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
Claims (15)
1. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za upotrebu u liječenju Fabryjeve bolesti kod pacijenta za kojeg je utvrđeno da ima reaktivni mutantni oblik α-Gal A, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija M1R, L14P, L16H, L16P, L19P, D33Y, N34K, G35R, L36F, A37V, P40L, P40S, M42L, M42R, M42T, L45R, W47L, E48D, E48K, R49G, R49L, R49P, R49S, M51I, C52R, L54P, D55V, C56F, C56G, C56Y, P60L, E66G, E66K, L68F, M72R, A73V, M76R, W81C, W81S, G85D, G85M, Y88D, C94Y, W95S, A97P, R112S, I117S, R118C, L120P, A121T, A135V, D136H, G147R, Y152C, A156T, A156Y, W162G, G163V, K168N, F169S, G171C, G171R, C172S, G183A, G183S, Y184C, M187T, M187V, L191P, L191Q, V199M, S201Y, P205L, P205R, P205S, N215D, Y216C, Y216D, I219N, N224D, N224S, H225R, A230T, D234E, W236L, S238N, I239T, I242N, L243F, L243W, D244H, S247C, S247P, Q250P, I253T, A257P, P259L, G261D, D264Y P265L, P265R, M267I, L268S, V269A, V269M, I270T, G271S, N272K, N272S, S276N, Q279R, Q280H, Q280K, A288D, A288P, A292P, P293A, P293S, P293T, S297C, N298H, N298K, N298S, D299G, L300F, R301G, R301P, I303N, A309P, L310F, D313G, I317N, I317T, N320I, Q321E, Q321L, Q321R, G325S, Q327E, E338K, V339E, W340R, E341D, S345P, A348P, A352D, I354K, E358G, I359T, G360D, G360S, G361R, P362L, R363P, E398K, P409S, P409T, T410A, T410I, T410P, G411D, L414S, 254del1, 247ins8, D55V/Q57L, i 401ins/T401S.
2. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema patentnom zahtjevu 1, naznačen time što je 1-deoksigalaktonojirimicin hidroklorid.
3. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema patentnom zahtjevu 1 ili zahtjevu 2, naznačen time što je pacijent žensko.
4. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema patentnom zahtjevu 1 ili zahtjevu 2, naznačen time što je pacijent muško.
5. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-4, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija D33Y, N34K, G35R, L36F, A37V, M42L, M42R, M42T, M51I, L54P, D55V, C56F, C56Y, P60L, E66G, E66K, A73V, G85D, G85M, A97P, R118C, A121T, A135V, Y152C, A156T, W162G, F169S, G183A, Y184C, M187T, M187V, L191Q, V199M, S201Y, P205L, P205S, N215D, Y216C, Y216D, I219N, N224S, S238N, I239T, I242N, L243F, L243W, D244H, S247C, Q250P, I253T, A257P, P259L, D264Y, P265L, V269A, V269M, I270T, G271S, S276N, Q280H, Q280K, A288P, P293T, N298S, L300F, R301G, R301P, I303N, A309P, L310F, D313G, I317T, N320I, Q321L, Q321R, G325S, Q327E, E338K, V339E, S345P, I354K, E358G, I359T, G360D, G360S, P362L, E398K, P409S, P409T, T410A, T410I, G411D, 254del1, D55V/Q57L, i 401ins/T401S.
6. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija D33Y, N34K, G35R, L36F, A37V, M42L, M42R, M42T, M51I, i L54P.
7. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija D55V, C56F, C56Y, P60L, E66G, E66K, A73V, G85D, G85M, i A97P.
8. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija R118C, A121T, A135V, Y152C, A156T, W162G, F169S, G183A, i Y184C.
9. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija M187T, M187V, L191Q, V199M, S201Y, P205L, P205S, N215D, Y216C, i Y216D.
10. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija I219N, N224S, S238N, I239T, I242N, L243F, L243W, D244H, i S247C.
11. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija Q250P, I253T, A257P, P259L, D264Y, P265L, V269A, V269M, i I270T.
12. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija G271S, S276N, Q280H, Q280K, A288P, P293T, N298S, L300F, R301G, i R301P.
13. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija I303N, A309P, L310F, D313G, I317T, N320I, Q321L, Q321R, G325S, i Q327E.
14. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija E338K, V339E, S345P, I354K, E358G, I359T, G360D, G360S, i P362L.
15. 1-deoksigalaktonojirimicin ili njegova farmaceutski prihvatljiva sol za uporabu prema bilo kojem od patentnih zahtjeva 1-5, naznačen time što je reaktivni mutantni oblik α-Gal A odabran od mutacija E398K, P409S, P409T, T410A, T410I, G411D, 254del1, D55V/Q57L, i 401ins/T401S.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US2814108P | 2008-02-12 | 2008-02-12 | |
US3568408P | 2008-03-11 | 2008-03-11 | |
US9363108P | 2008-09-02 | 2008-09-02 | |
US11349608P | 2008-11-11 | 2008-11-11 | |
EP18201960.4A EP3470077B1 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
Publications (1)
Publication Number | Publication Date |
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HRP20201827T1 true HRP20201827T1 (hr) | 2021-01-08 |
Family
ID=40957500
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20150728TT HRP20150728T1 (hr) | 2008-02-12 | 2015-07-06 | Postupak za predviđanje reakcije na liječenje bolesti s farmakološkim šaperonima |
HRP20190143TT HRP20190143T1 (hr) | 2008-02-12 | 2019-01-22 | Postupak za predviđanje odgovora na liječenje bolesti farmakološkim šaperonom |
HRP20201827TT HRP20201827T1 (hr) | 2008-02-12 | 2020-11-17 | Postupak za predviđanje odgovora na liječenje bolesti farmakološkim šaperonom |
Family Applications Before (2)
Application Number | Title | Priority Date | Filing Date |
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HRP20150728TT HRP20150728T1 (hr) | 2008-02-12 | 2015-07-06 | Postupak za predviđanje reakcije na liječenje bolesti s farmakološkim šaperonima |
HRP20190143TT HRP20190143T1 (hr) | 2008-02-12 | 2019-01-22 | Postupak za predviđanje odgovora na liječenje bolesti farmakološkim šaperonom |
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US (7) | US8592362B2 (hr) |
EP (4) | EP3824900A1 (hr) |
JP (6) | JP5844045B2 (hr) |
AU (6) | AU2009214648B2 (hr) |
CA (1) | CA2715407C (hr) |
CY (3) | CY1116466T1 (hr) |
DK (3) | DK3470077T3 (hr) |
ES (3) | ES2716502T3 (hr) |
HR (3) | HRP20150728T1 (hr) |
HU (3) | HUE051377T2 (hr) |
LT (2) | LT2946785T (hr) |
MX (1) | MX2010008835A (hr) |
PL (3) | PL2946785T3 (hr) |
PT (3) | PT3470077T (hr) |
SI (3) | SI2252313T1 (hr) |
WO (1) | WO2009102895A2 (hr) |
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GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
LT3397273T (lt) | 2015-12-30 | 2021-06-25 | Amicus Therapeutics, Inc. | Patobulinta rūgštinė alfa-gliukozidazė pompe ligos gydymui |
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CN109475607B (zh) | 2016-03-30 | 2022-04-26 | 阿米库斯治疗学公司 | 包含重组酸性α-葡糖苷酶的配制品 |
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