CN113365640A - 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白 - Google Patents

引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白 Download PDF

Info

Publication number
CN113365640A
CN113365640A CN201980080522.8A CN201980080522A CN113365640A CN 113365640 A CN113365640 A CN 113365640A CN 201980080522 A CN201980080522 A CN 201980080522A CN 113365640 A CN113365640 A CN 113365640A
Authority
CN
China
Prior art keywords
therapeutic agent
patient
level
biological sample
neurofilament
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN201980080522.8A
Other languages
English (en)
Chinese (zh)
Inventor
T.A.弗古森
D.L.格雷厄姆
S.S-W.韩
A.R.V.麦坎贝尔
G.斯鲁贝克托马西
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biogan Ma Co
Original Assignee
Biogan Ma Co
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 Biogan Ma Co filed Critical Biogan Ma Co
Publication of CN113365640A publication Critical patent/CN113365640A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6887Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids from muscle, cartilage or connective tissue
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y115/00Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001Superoxide dismutase (1.15.1.1)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Analytical Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Food Science & Technology (AREA)
CN201980080522.8A 2018-12-06 2019-12-03 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白 Pending CN113365640A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862776253P 2018-12-06 2018-12-06
US62/776,253 2018-12-06
US201962840431P 2019-04-30 2019-04-30
US62/840,431 2019-04-30
US201962842063P 2019-05-02 2019-05-02
US62/842,063 2019-05-02
PCT/US2019/064190 WO2020117772A1 (en) 2018-12-06 2019-12-03 Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
CN113365640A true CN113365640A (zh) 2021-09-07

Family

ID=68966114

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980080522.8A Pending CN113365640A (zh) 2018-12-06 2019-12-03 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白

Country Status (10)

Country Link
US (1) US12429487B2 (https=)
EP (1) EP3890752A1 (https=)
JP (2) JP2022513721A (https=)
KR (1) KR20210102294A (https=)
CN (1) CN113365640A (https=)
BR (1) BR112021010660A2 (https=)
CA (1) CA3121749A1 (https=)
MA (1) MA54383A (https=)
MX (1) MX2021006548A (https=)
WO (1) WO2020117772A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119679944A (zh) 2018-01-25 2025-03-25 渤健马萨诸塞州股份有限公司 治疗脊髓性肌萎缩症的方法
US12429487B2 (en) 2018-12-06 2025-09-30 Biogen Ma Inc. Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis
CN114667597A (zh) 2019-06-11 2022-06-24 库力索法荷兰有限公司 在分立部件的组装中用于位置误差补偿的材料
WO2022091086A1 (en) * 2020-10-26 2022-05-05 Hadasit Medical Research Services And Development Ltd. Mesenchymal stem cells and their culture
KR200498472Y1 (ko) 2024-05-23 2024-10-24 김민경 경고 또는 안내 문구를 교체형으로 구성시킬 수 있는 배수용 그레이팅

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019915A1 (en) * 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
CN104853778A (zh) * 2012-10-24 2015-08-19 第一三共株式会社 用于肌萎缩性侧索硬化的治疗剂
WO2015153800A2 (en) * 2014-04-01 2015-10-08 Isis Pharmaceuticals, Inc. Compositions for modulating sod-1 expression
CN107109407A (zh) * 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2221376B1 (en) 2001-06-21 2012-11-21 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
WO2008127974A1 (en) 2007-04-13 2008-10-23 The Trustees Of Columbia University In The City Of New York Stem cell-based culture system for drug development
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
US8465727B2 (en) 2009-10-06 2013-06-18 University of Pittsburgh—of the Commonwealth System of Higher Education Biomarkers for the diagnosis of ALS
WO2013170068A2 (en) 2012-05-09 2013-11-14 Karyopharm Therapeutics, Inc. Nuclear transport modulators and uses thereof
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
CN104968783B (zh) 2012-10-15 2019-12-10 Ionis制药公司 用于调节c9orf72表达的组合物
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10221414B2 (en) 2013-10-11 2019-03-05 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
WO2015057727A1 (en) 2013-10-14 2015-04-23 Isis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
CA2926341A1 (en) 2013-10-14 2015-04-23 Ionis Pharmaceuticals, Inc. Methods for modulating expression of c9orf72 antisense transcript
EP3119888B1 (en) 2014-03-19 2021-07-28 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
US10024870B2 (en) 2014-04-02 2018-07-17 Wisconsin Alumni Research Foundation Therapeutic and diagnostic methods and compositions for neurodegenerative diseases
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
SG10201902850TA (en) 2014-09-30 2019-04-29 Neurimmune Holding Ag Human-derived anti-dipeptide repeats (dprs) antibody
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
RS60230B1 (sr) 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
US20190142856A1 (en) 2016-04-13 2019-05-16 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
WO2018218219A1 (en) 2017-05-26 2018-11-29 University Of Miami Determining onset of amyotrophic lateral sclerosis
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
CN119679944A (zh) 2018-01-25 2025-03-25 渤健马萨诸塞州股份有限公司 治疗脊髓性肌萎缩症的方法
EA201992364A1 (ru) 2018-04-02 2020-03-23 Кохерус Байосайенсис Инк. АГОНИСТ PPARγ ДЛЯ ЛЕЧЕНИЯ ПРОГРЕССИРУЮЩЕГО НАДЪЯДЕРНОГО ПАРАЛИЧА
US12162952B2 (en) 2018-04-27 2024-12-10 Neurimmune Ag Human-derived anti-(poly-GA) dipeptide repeat (DPR) antibody
WO2020061355A2 (en) 2018-09-20 2020-03-26 Biogen Ma Inc. Serum neurofilament protein for guiding therapeutic intervention in multiple sclerosis
US12429487B2 (en) 2018-12-06 2025-09-30 Biogen Ma Inc. Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis
TWI906204B (zh) 2018-12-14 2025-12-01 美商百健Ma公司 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法
US20220128578A1 (en) 2019-02-12 2022-04-28 Biogen Ma Inc. Biomarkers of progressive multifocal leukoencephalopathy

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019915A1 (en) * 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
CN104853778A (zh) * 2012-10-24 2015-08-19 第一三共株式会社 用于肌萎缩性侧索硬化的治疗剂
WO2015153800A2 (en) * 2014-04-01 2015-10-08 Isis Pharmaceuticals, Inc. Compositions for modulating sod-1 expression
CN106459972A (zh) * 2014-04-01 2017-02-22 Ionis制药公司 用于调节sod‑1表达的组合物
CN107109407A (zh) * 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
MICHAEL BENATAR ET AL.: ""Neurofilament light: A candidate biomarker of presymptomatic amyotrophic lateral sclerosis and phenoconversion: Neurofilament Light in Presymptomatic ALS"", 《ANNALS OF NEUROLOGY》, vol. 84, no. 1, pages 130 - 139, XP055669467, DOI: 10.1002/ana.25276 *
ROSENGREN L E ETAL.: "\"Patients with amyotrophic lateral sclerosis and other neurodegenerative diseases have increased levels of neurofilament protein in csf\"", 《JOURNAL OF NEUROCHEMISTRYWILEY-BLACKWELL》, vol. 67, no. 51, pages 2013 - 2018, XP009075558 *
ROSSI D, ET AL.: ""CSF neurofilament proteins as diagnostic and prognostic biomarkers for amyotrophic lateral sclerosis"", 《JOURNAL OF NEUROLOGY》, vol. 265, pages 510 - 521, XP036445948, DOI: 10.1007/s00415-017-8730-6 *

Also Published As

Publication number Publication date
JP2024096258A (ja) 2024-07-12
BR112021010660A2 (pt) 2021-08-24
US12429487B2 (en) 2025-09-30
JP2022513721A (ja) 2022-02-09
MX2021006548A (es) 2021-09-21
CA3121749A1 (en) 2020-06-11
US20220034907A1 (en) 2022-02-03
WO2020117772A1 (en) 2020-06-11
KR20210102294A (ko) 2021-08-19
MA54383A (fr) 2021-10-13
EP3890752A1 (en) 2021-10-13

Similar Documents

Publication Publication Date Title
CN113365640A (zh) 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白
DK2367849T3 (en) METHOD OF TREATING NEURODEGENERATIVE DISEASES
Todd et al. Correcting N euromuscular D eficits W ith G ene T herapy in P ompe D isease
US20250230440A1 (en) Methods of treating or preventing spinal muscular atrophy
KR102821047B1 (ko) 근위축 측삭 경화증의 치료 및 방지를 위한 조성물 및 방법
CA3101321A1 (en) Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy
Corrà et al. Double administration of self-complementary AAV9 NDUFS4 prevents Leigh disease in Ndufs4−/− mice
Lai et al. Increase of HCN current in SOD1-associated amyotrophic lateral sclerosis
RU2844865C2 (ru) Нейрофиламентный белок для направления терапевтических вмешательств при боковом амиотрофическом склерозе
Kwon et al. Analyzing clinical and genetic aspects of axonal Charcot–Marie-Tooth disease
Evans-Galea et al. A novel deletion–insertion mutation identified in exon 3 of FXN in two siblings with a severe Friedreich ataxia phenotype
US20240182903A1 (en) Treatment of amyotrophic lateral sclerosis
Sar Analysis of the therapeutic strategies used to treat Amyotrophic Lateral Sclerosis caused by TDP-43 aggregation and/or mislocalization
US20230028590A1 (en) Als treatment method, als pharmaceutical composition, and als diagnostic method
RU2824046C2 (ru) Составы для лечения и предотвращения бокового амиотрофического склероза
WO2013184209A1 (en) Mif for use in methods of treating subjects with a neurodegenerative disorder
Quilgars et al. Brief early-life motor training induces behavioral changes and alters neuromuscular development in mice
EA047168B1 (ru) Способы лечения или предотвращения спинальной мышечной атрофии
WO2026043810A1 (en) Manipulating golgi ions for treatment of neurodegenerative disease and cutaneous disorders
WO2026025082A1 (en) Methods for treating alzheimer's disease
Vincent O07 The John Newsom-Davis Lecture: The neuromuscular junction–a wide spectrum of disease mechanisms
EA051069B1 (ru) Составы и способы лечения и предотвращения бокового амиотрофического склероза

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination