[go: up one dir, main page]

SG11201903917WA - Inhibitors of gangliosides metabolism for the treatment of motor neuron diseases - Google Patents

Inhibitors of gangliosides metabolism for the treatment of motor neuron diseases

Info

Publication number
SG11201903917WA
SG11201903917WA SG11201903917WA SG11201903917WA SG11201903917WA SG 11201903917W A SG11201903917W A SG 11201903917WA SG 11201903917W A SG11201903917W A SG 11201903917WA SG 11201903917W A SG11201903917W A SG 11201903917WA SG 11201903917W A SG11201903917W A SG 11201903917WA
Authority
SG
Singapore
Prior art keywords
paris
international
rule
inhibitors
avenue
Prior art date
Application number
SG11201903917WA
Inventor
Frédéric Darios
Giovanni Stevanin
Fanny Mochel
Julien Branchu
Maxime Boutry
Original Assignee
Icm Inst Du Cerveau Et De La Moelle Epiniere
Univ Sorbonne
Centre Nat Rech Scient
Inst Nat Sante Rech Med
Aphp Assist Publique Hopitaux De Paris
Ecole Pratique Des Hautes Etudes
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Icm Inst Du Cerveau Et De La Moelle Epiniere, Univ Sorbonne, Centre Nat Rech Scient, Inst Nat Sante Rech Med, Aphp Assist Publique Hopitaux De Paris, Ecole Pratique Des Hautes Etudes filed Critical Icm Inst Du Cerveau Et De La Moelle Epiniere
Publication of SG11201903917WA publication Critical patent/SG11201903917WA/en

Links

Classifications

    • 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
    • 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
    • A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • 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
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/137—Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine or methadone
    • 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
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/4025—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil not condensed and containing further heterocyclic rings, e.g. cromakalim
    • 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
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A61P25/10—Antiepileptics; Anticonvulsants for petit-mal
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Emergency Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111111111111111111111111111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\" WO 2018/083223 Al 11 May 2018 (11.05.2018) WIPO I PCT (51) International Patent Classification: UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, A61K 45/06 (2006.01) A61P 25/10 (2006.01) TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, A61K 31/445 (2006.01) EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, (21) International Application Number: MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, PCT/EP2017/078156 TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (22) International Filing Date: 03 November 2017 (03.11.2017) Declarations under Rule 4.17: — of inventorship (Rule 4.17(iv)) (25) Filing Language: English (26) Publication Language: English Published: — with international search report (Art. 21(3)) (30) Priority Data: — before the expiration of the time limit for amending the 16197362.3 04 November 2016 (04.11.2016) EP claims and to be republished in the event of receipt of (71) Applicants: ICM (INSTITUT DU CERVEAU ET amendments (Rule 48.2(h)) — DE LA MOELLE EPINIERE) [FR/FR]; Hopital Pitie with sequence listing part of description (Rule 5.2(a)) Salpetriere, 47/83 boulevard de l'Hopital, 75013 Paris (FR). UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6 (UPMC) [FR/FR]; 4 place Jussieu, 75005 Paris (FR). CENTRE NATIONAL DE LA RECHERCHE SCIEN- _ TIFIQUE [FR/FR]; 3 me Michel-Ange, 75016 Paris (FR). INSERM (INSTITUT NATIONAL DE LA SAN- — TE ET DE LA RECHERCHE MEDICALE) [FR/FR]; _ 101 rue de Tolbiac, 75654 Paris Cedex 13 (FR). APHP = (ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS) [FR/FR]; 3 avenue Victoria, 75001 Paris (FR). ECOLE PRATIQUE DES HAUTES ETUDES [FR/FR]; Les Pa- tios Saint Jacques, 4-14 me Ferrus, 75014 Paris (FR). Inventors: DARIOS, Frederic; 22 me Esther, 92160 = (72) Antony (FR). STEVANIN, Giovanni; 19 avenue Vic- tor Hugo, 93270 Sevran (FR). MOCHEL, Fanny; 55 me Georges Lardennois, 75019 Paris (FR). BRANCHU, Julien; 75 me Vauvenargues, 75018 Paris (FR). BOUTRY, Maxime; 44 me Coriolis, 75012 Paris (FR). _ = = Agent: ICOSA; 83 avenue Denfert-Rochereau, 75014 Paris (74) (FR). = Designated States (unless otherwise indicated, for every (81) = kind of national protection available): AE, AG, AL, AM, = AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, = — CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, = DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = — 1-1 N el (84) Designated States (unless otherwise indicated, for every N en GC kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, 0 --.... 00 1-1 (54) Title: INHIBITORS OF GANGLIOSIDES METABOLISM FOR THE TREATMENT OF MOTOR NEURON DISEASES 0 ei (57) : The present invention relates to inhibitors of gangliosides metabolism for treating motor neuron diseases, in particular C hereditary spastic paraplegias.
SG11201903917WA 2016-11-04 2017-11-03 Inhibitors of gangliosides metabolism for the treatment of motor neuron diseases SG11201903917WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP16197362.3A EP3318277A1 (en) 2016-11-04 2016-11-04 Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases
PCT/EP2017/078156 WO2018083223A1 (en) 2016-11-04 2017-11-03 Inhibitors of gangliosides metabolism for the treatment of motor neuron diseases

Publications (1)

Publication Number Publication Date
SG11201903917WA true SG11201903917WA (en) 2019-05-30

Family

ID=57249710

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201903917WA SG11201903917WA (en) 2016-11-04 2017-11-03 Inhibitors of gangliosides metabolism for the treatment of motor neuron diseases

Country Status (10)

Country Link
US (2) US11065238B2 (en)
EP (2) EP3318277A1 (en)
JP (1) JP7164537B2 (en)
KR (1) KR20190088475A (en)
CN (1) CN110121361A (en)
AU (1) AU2017353446B2 (en)
CA (1) CA3042587A1 (en)
IL (1) IL266389A (en)
SG (1) SG11201903917WA (en)
WO (1) WO2018083223A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7302871B2 (en) 2016-04-21 2023-07-04 ベイラー カレッジ オブ メディスン Compositions and methods for treating lysosomal storage disorders and disorders characterized by lysosomal dysfunction
JP7725076B2 (en) 2020-04-28 2025-08-19 ザ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・ミシガン Pyridine inhibitors of glucosylceramide synthase and methods of treatment using them
CN117904285A (en) * 2024-03-19 2024-04-19 北京诺禾致源科技股份有限公司 Application of GBA2 mutant gene as biomarker in preparation of product for diagnosing autosomal recessive spastic paraplegia 46

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE2834122A1 (en) 1978-08-03 1980-02-14 Bayer Ag METHOD FOR PRODUCING 6-AMINO-6-DESOXY-L-SORBOSE
DE2853573A1 (en) 1978-12-12 1980-07-03 Bayer Ag PREPARATION OF N-SUBSTITUTED DERIVATIVES OF L-DESOXYNOJIRIMYCIN
DE3038901A1 (en) 1980-10-15 1982-05-06 Bayer Ag, 5090 Leverkusen METHOD FOR PRODUCING N-SUBSTITUTED DERIVATIVES OF 1-DESOXYNOJIRIMYCIN
GB8308235D0 (en) 1983-03-25 1983-05-05 Celltech Ltd Polypeptides
US5225539A (en) 1986-03-27 1993-07-06 Medical Research Council Recombinant altered antibodies and methods of making altered antibodies
DE3610997A1 (en) 1986-04-02 1987-10-15 Krewel Werke Gmbh AMBROXOL NOSE SPRAY
DE3611841A1 (en) 1986-04-09 1987-10-15 Bayer Ag METHOD FOR PRODUCING 1-DESOXYNOJIRIMYCIN AND ITS N-DERIVATIVES
US4946778A (en) 1987-09-21 1990-08-07 Genex Corporation Single polypeptide chain binding molecules
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US5043273A (en) 1989-08-17 1991-08-27 Monsanto Company Phosphorylated glycosidase inhibitor prodrugs
US4994572A (en) 1989-10-12 1991-02-19 Monsanto Company Synthesis of nojirimycin derivatives
US5200523A (en) 1990-10-10 1993-04-06 Monsanto Company Synthesis of nojirimycin derivatives
US5258518A (en) 1992-04-01 1993-11-02 G. D. Searle & Co. 2-substituted tertiary carbinol derivatives of deoxynojirimycin
US5302609A (en) 1992-12-16 1994-04-12 The Regents Of The University Of Michigan Treatment of diabetic nephropathy
US5399567A (en) 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US5798366A (en) * 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
AU1876095A (en) 1994-02-25 1995-09-11 G.D. Searle & Co. Use of 1-deoxynojirimycin and its derivatives for treating mammals infected with respiratory syncytial virus
US6255336B1 (en) 1995-09-20 2001-07-03 The Regents Of The University Of Michigan Amino ceramide-like compounds and therapeutic methods of use
US5916911A (en) 1995-09-20 1999-06-29 The Regents Of The University Of Michigan Amino ceramide--like compounds and therapeutic methods of use
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
AUPP249298A0 (en) 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
US6566131B1 (en) 2000-10-04 2003-05-20 Isis Pharmaceuticals, Inc. Antisense modulation of Smad6 expression
US6410323B1 (en) 1999-08-31 2002-06-25 Isis Pharmaceuticals, Inc. Antisense modulation of human Rho family gene expression
US6107091A (en) 1998-12-03 2000-08-22 Isis Pharmaceuticals Inc. Antisense inhibition of G-alpha-16 expression
US5981732A (en) 1998-12-04 1999-11-09 Isis Pharmaceuticals Inc. Antisense modulation of G-alpha-13 expression
US6610703B1 (en) 1998-12-10 2003-08-26 G.D. Searle & Co. Method for treatment of glycolipid storage diseases
US6046321A (en) 1999-04-09 2000-04-04 Isis Pharmaceuticals Inc. Antisense modulation of G-alpha-i1 expression
US6280989B1 (en) 1999-06-17 2001-08-28 Dmitri Kapitonov Sialyltransferases
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
JP2003526367A (en) 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド RNA interference method and RNA interference composition
US6365354B1 (en) 2000-07-31 2002-04-02 Isis Pharmaceuticals, Inc. Antisense modulation of lysophospholipase I expression
US6566135B1 (en) 2000-10-04 2003-05-20 Isis Pharmaceuticals, Inc. Antisense modulation of caspase 6 expression
US6916802B2 (en) 2002-04-29 2005-07-12 Genzyme Corporation Amino ceramide-like compounds and therapeutic methods of use
EP1528056A1 (en) 2003-10-29 2005-05-04 Academisch Ziekenhuis bij de Universiteit van Amsterdam Deoxynojirimycin analogues and their uses as glucosylceramidase inhibitors
JP4767019B2 (en) 2004-01-16 2011-09-07 武田薬品工業株式会社 Pharmaceuticals for prevention and treatment of arteriosclerosis
MXPA06012690A (en) 2004-05-07 2007-01-16 Merck Patent Gmbh Gm3 synthase as a therapeutic target in microvascular complications of diabetes.
EP2023927B1 (en) 2006-05-24 2015-02-25 United Therapeutics Corporation Deoxynojirimycin and d-arabinitol analogs and methods of using
DK1860101T3 (en) 2006-05-24 2011-07-25 Amicus Therapeutics Inc Tartrate salt of isophagomine and methods for its use
WO2010091104A1 (en) 2009-02-06 2010-08-12 Exelixis, Inc. Glucosylceramide synthase inhibitors
WO2010091164A1 (en) 2009-02-06 2010-08-12 Exelixis, Inc. Inhibitors of glucosylceramide synthase
MX381242B (en) 2009-11-27 2025-03-12 Genzyme Corp AN AMORPHOUS AND CRYSTALLINE FORM OF GENZ HEMITARTRATE 112638 AS AN INHIBITOR OF GLUCOSYLCERAMIDE SYNTHASE.
GB201000499D0 (en) 2010-01-13 2010-03-03 Fleet George W J Treatment of cystic fibrosis
WO2011095772A2 (en) 2010-02-04 2011-08-11 Summit Corporation Plc Novel iminosugar therapeutics
WO2011133918A1 (en) 2010-04-23 2011-10-27 Isis Pharmaceuticals, Inc. Modulation of gm3 synthase (gm3s) expression
GB201103526D0 (en) 2011-03-02 2011-04-13 Summit Corp Plc Selective glycosidase inhibitors and uses thereof
LT3673906T (en) 2011-03-18 2025-08-11 Genzyme Corporation Glucosylceramide synthase inhibitors
US9845327B2 (en) * 2011-06-22 2017-12-19 The General Hospital Corporation Treatment of proteinopathies
EP2739304A4 (en) * 2011-08-04 2015-06-03 Univ Ramot Particles for the treatment of neurodegenerative diseases
US8961959B2 (en) 2011-10-17 2015-02-24 The Regents Of The University Of Michigan Glucosylceramide synthase inhibitors and therapeutic methods using the same
JOP20130273B1 (en) 2012-09-11 2021-08-17 Genzyme Corp Glucosylceramide synthase inhibitors
EP3122726B1 (en) 2014-03-27 2018-08-29 Academisch Medisch Centrum N-(5-((aryl or heteroaryl)methyloxy)pentyl)-substituted iminosugars as inhibitors of glucosylceramide synthase
EP3442514B1 (en) * 2016-04-11 2021-06-16 Spedding Research Solutions Inhibitors of glucosylceramide degradation in the treatment of diseases of the motor units

Also Published As

Publication number Publication date
CN110121361A (en) 2019-08-13
US20220062254A1 (en) 2022-03-03
EP3534958A1 (en) 2019-09-11
US11065238B2 (en) 2021-07-20
EP3318277A1 (en) 2018-05-09
AU2017353446A1 (en) 2019-05-23
JP7164537B2 (en) 2022-11-01
AU2017353446B2 (en) 2021-04-29
IL266389A (en) 2019-06-30
CA3042587A1 (en) 2018-05-11
WO2018083223A1 (en) 2018-05-11
JP2020500216A (en) 2020-01-09
US20190350913A1 (en) 2019-11-21
KR20190088475A (en) 2019-07-26

Similar Documents

Publication Publication Date Title
SG11201901494UA (en) Acid-alpha glucosidase variants and uses thereof
SG11201811431VA (en) Multispecific antibodies against cd40 and cd137
SG11201900845YA (en) Uses of il-13 antagonists for treating atopic dermatitis
SG11201806340YA (en) Zika virus vaccine
SG11201807778YA (en) Bicyclic compounds for diagnosis and therapy
SG11201906297QA (en) Nucleic acids encoding crispr-associated proteins and uses thereof
SG11201807827VA (en) Process for the preparation of pegylated drug-linkers and intermediates thereof
SG11201903697WA (en) Liver organoid compositions and methods of making and using same
SG11201808222RA (en) Methods of treatment of cholestatic diseases
SG11201801401UA (en) A pharmaceutical composition for use in the treatment or prevention of a c5-related disease and a method for treating or preventing a c5-related disease
SG11201811491YA (en) Quinazoline and indole compounds to treat medical disorders
SG11201908325PA (en) Process for the preparation of glucuronide drug-linkers and intermediates thereof
SG11201804510PA (en) Biparatopic polypeptides antagonizing wnt signaling in tumor cells
SG11201901063SA (en) Therapeutic agents for neurodegenerative diseases
SG11201901961SA (en) Methods of using a bispecific antibody that recognizes coagulation factor ix and/or activated coagulation factor ix and coagulation factor x and/or activated coagulation factor x
SG11201805195YA (en) Composition comprising botulinum toxin
SG11201907583TA (en) Methods for treating complement-mediated diseases and disorders
SG11201809875VA (en) Compositions and methods for treating spinal muscular atrophy
SG11201807041PA (en) Improved preparations of adult liver progenitor cells
SG11201809531SA (en) BINDING MOLECULES SPECIFIC FOR FCγGAMMA RIIA AND USES THEREOF
SG11201809534UA (en) Methods of treating autoimmune disease using allogeneic t cells
SG11201805311XA (en) Method of treating a mammal, including human, against cancer using methionine and asparagine depletion
SG11201805001UA (en) Method of treating influenza a
SG11201808829VA (en) Tdp-43 mitochondrial localization inhibitor for the treatment of neurodegenerative disease
SG11201805649UA (en) Therapeutic anti-cd9 antibody