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2025
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Invention
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Treatment of genetic cardiomyopathies with aav gene therapy vectors. Provided herein are gene the... |
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Invention
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C-type natriuretic peptide variants to treat skeletal dysplasia in children.
The present disclos... |
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2024
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P/S
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Pharmaceutical preparations for use in enzyme therapy;
pharmaceutical preparations for use in tr... |
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Invention
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Conjugates of phenylalanine ammonia-lyase and poegma and methods for their use.
The present disc... |
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Invention
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Conjugates of phenylalanine ammonia-lyase and poegma and methods for their use. The present discl... |
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Invention
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Cnp variants, conjugates and formulations thereof. Provided herein are pharmaceutical composition... |
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Invention
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Treatment of osteogenesis imperfecta with c-type natriuretic peptide and analogs thereof. The pre... |
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P/S
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Downloadable computer application software for mobile
phones, namely, software for patients to a... |
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Invention
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Dosage forms of n-(1-(2-chloro-3-fluorophenyl)-1-hydroxypentan-2-yl)-7-fluoro-2-oxoindoline-4-car... |
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Invention
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Solid forms of n-1-(2-chloro-3-fluorophenyl)-1-hydroxypentan-2-yl)-7-fluoro-2-oxoindoline-4-carbo... |
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Invention
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Methods of manufacturing highly stereochemically pure inhibitors of z-at polymerization. 122 is p... |
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Invention
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Use of c-type natriuretic peptide variants to treat skeletal dysplasia.
The present disclosure p... |
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P/S
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Maintaining patient medical records and files Pharmaceutical preparations for use in enzyme thera... |
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Invention
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High throughput screen for genetic variants associated with short stature. The present disclosure... |
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Invention
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Analytical methods for adeno-associated virus vector genomes and uses thereof. Methods of analyzi... |
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Invention
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Adeno-associated virus capsid proteins.
The invention relates to novel adeno-associated virus (A... |
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Invention
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Methods of treating anti-aav seropositive hemophilia patients. The disclosure relates to methods ... |
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Invention
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Stable expression of aav vectors in juvenile subjects.
The invention relates to the use of adeno... |
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Invention
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Dystrophin exon skipping oligonucleotides. Provided herein are antisense oligonucleotides (ASOs) ... |
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Invention
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Dystrophin exon skipping oligonucleotides.
Provided herein are antisense oligonucleotides (ASOs)... |
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Invention
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Compositions and methods for treating stxbp1 disorders. Provided herein are antisense oligonucleo... |
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P/S
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Downloadable computer application software for mobile phones, namely, software for patients to ac... |
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Invention
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Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof. ... |
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P/S
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Pharmaceutical preparations for treating genetic diseases and disorders. |
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Invention
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Epigenetic modifiers improve aav gene therapy durability. The disclosure provides for methods of ... |
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2023
|
Invention
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Methods of redosing gene therapy vectors.
The present disclosure relates, in general, to methods... |
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Invention
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Treatment of cardiomyopathy with aav gene therapy vectors. Provided herein are gene therapy compo... |
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Invention
|
Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors. Provided herein are gen... |
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|
Invention
|
Compositions and methods for treating long qt syndrome. The present disclosure provides anti-KCNQ... |
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|
Invention
|
Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulatio... |
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|
Invention
|
Method of treating duchenne muscular dystrophy.
Provided herein are methods of treating or delay... |
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|
Invention
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Method of treating duchenne muscular dystrophy. Provided herein are methods of treating or delayi... |
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|
Invention
|
Dystrophin exon skipping oligonucleotides.
Provided herein are antisense oligonucleotides (AONs)... |
|
|
Invention
|
Dystrophin exon skipping oligonucleotides. Provided herein are antisense oligonucleotides (AONs) ... |
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P/S
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Pharmaceutical preparations for treating genetic diseases and disorders |
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P/S
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pharmaceuticals. downloadable educational course materials. educational services. pharmaceutical ... |
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Invention
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Use of c-type natriuretic peptide variants to treat skeletal dysplasia. The present disclosure pr... |
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2022
|
Invention
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Novel adeno-associated virus capsid proteins.
The invention relates to novel adeno-associated vi... |
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Invention
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Cnp therapy.
The present disclosure relates, in general, to measures of efficacy in patients rec... |
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Invention
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C-type natriuretic peptide therapy of bone-related disorders. The present disclosure relates, in ... |
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|
Invention
|
Compositions of prokaryotic phenylalanine ammonia-lyase variants and methods of using composition... |
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|
Invention
|
Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations.
P... |
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|
Invention
|
Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations. Pr... |
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|
Invention
|
Aav capsid compositions and methods for delivery.
The disclosure provides various compositions c... |
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|
Invention
|
Lysosomal targeting peptides and uses thereof.
The present invention provides further improved c... |
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|
Invention
|
Large scale adeno-associated virus production systems.
The present invention provides processes ... |
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|
Invention
|
C-type natriuretic peptide variants to treat skeletal dysplasia in children. treatment of skeleta... |
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|
Invention
|
Compositions of beta-hexosaminidase variants and uses thereof.
Disclosed herein are recombinant ... |
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders |
|
|
Invention
|
Use of histidine rich peptides as a transfection reagent for raav and rbv production.
The presen... |
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Invention
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Aav production systems for aav viral particles with improved infectivity.
The present invention ... |
|
2021
|
Invention
|
Process for enriching adeno-associated virus.
The present invention provides process for enrichi... |
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders. |
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P/S
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Pharmaceutical preparations for the treatment of genetic
diseases and disorders. |
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2020
|
Invention
|
Treatment of hereditary angioedema with liver-specific gene therapy vectors.
Provided herein are... |
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P/S
|
Entertainment services in the nature of live theatrical performances; Educational services, namel... |
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P/S
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Medical information; providing health information; providing
medical information in the field of... |
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|
Invention
|
Cnp variants and conjugates thereof.
The present disclosure, relates, in general, to stable vari... |
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P/S
|
Medical information; Providing health information; Providing medical information in the field of ... |
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P/S
|
Pharmaceutical preparations for treating genetic diseases and disorders. |
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P/S
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Pharmaceutical preparations for the treatment of genetic diseases and disorders. |
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2019
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders; peptides for the... |
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P/S
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Providing medical information in the field of medical conditions giving rise to mucopolysaccharid... |
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P/S
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Medical information; providing health information; providing medical information in the field of ... |
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P/S
|
Pharmaceutical preparations for use in enzyme therapy; pharmaceutical preparations for treating g... |
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P/S
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Providing medical information in the field of genetic diseases and disorders, namely, genetic ske... |
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders |
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders; pharmaceutical p... |
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2018
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders; Pharmaceutical p... |
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P/S
|
Pharmaceutical preparations for the treatment of genetic diseases and disorders, namely, enzyme d... |
