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2024
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P/S
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Pharmaceutical preparations for the treatment of pompe disease; pharmaceutical preparations for t... |
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P/S
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Pharmaceutical preparations for the treatment of Pompe disease; Pharmaceutical preparations for t... |
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Invention
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Recombinant human acid alpha-glucosidase.
Provided are a recombinant acid α-glucosidase and phar... |
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Invention
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Gene therapy constructs for the treatment of pompe disease. The present disclosure relates to rec... |
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2023
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P/S
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Downloadable computer application software for mobile devices, namely, software for Pompe disease... |
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Invention
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Methods for treating late onset pompe disease in pediatric patients. Provided herein are methods ... |
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Invention
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Fexamethods for treating infantile-onset pompe disease in pediatric patients. Provided herein are... |
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Invention
|
Methods of treating fabry disease in patients having a mutation in the gla gene.
Provided are me... |
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P/S
|
Downloadable computer application software for mobile
devices, namely, software for pompe diseas... |
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P/S
|
Downloadable computer application software for mobile devices, namely, software for pompe disease... |
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Invention
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Dosing regimens for the treatment of fabry disease.
The presently disclosed subject matter provi... |
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Invention
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Highly purified batches of pharmaceutical grade migalastat and methods of producing the same. Pro... |
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Invention
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Methods of improving the pharmacokinetics of migalastat. Provided are methods of improving the ph... |
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Invention
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Methods of improving the pharmacokinetics of migalastat. Provided are methods of improving ... |
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Invention
|
Methods of treating fabry patients having renal impairment. Provided are methods for treatment of... |
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Invention
|
Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones.... |
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Invention
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Treatment of fabry disease in ert-naïve and ert-experienced patients.
Provided are dosing regime... |
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Invention
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Methods of improving the pharmacokinetics of migalastat.
Provided are methods of improving the p... |
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Invention
|
Methods for treating pompe disease |
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|
Invention
|
Methods for treating pompe disease. Provided herein are methods for treating Pompe disease by adm... |
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P/S
|
Providing medical services for the treatment of rare genetic diseases; Providing health informati... |
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Invention
|
Highly potent acid alpha-glucosidase with enhanced carbohydrates.
Recombinant human alpha glucos... |
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2022
|
Invention
|
Compositions and methods for treating mucopolysaccharidosis iiia.
Provided herein are compositio... |
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Invention
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Gene therapy for treatment of mucopolysaccharidosis iiia. Provided herein is a recombinant AAV (r... |
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Invention
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Compositions and methods for treating mucopolysaccharidosis iiia. Provided herein are composition... |
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Invention
|
Formulations comprising recombinant acid alpha-glucosidase.
Provided are pharmaceutical formulat... |
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Invention
|
Gene therapy constructs and methods of use.
Provided herein are improved gene therapy vectors an... |
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Invention
|
Compositions useful in treatment of cdkl5 deficiency disorder (cdd). Provided is a recombinant ad... |
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Invention
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Biomarkers for lysosomal storage diseases.
Methods of determining a disease score of a patient d... |
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|
Invention
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Biomarkers for lysosomal storage diseases. Methods of determining a disease score of a patient di... |
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Invention
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Method for selection of high m6p recombinant proteins.
Methods for the production, capturing and... |
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Invention
|
Determination of gene transduction potency in neuron-like cells. The disclosure provides a method... |
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Invention
|
Methods of treating fabry disease in pediatric patients.
Provided are methods for the treatment ... |
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Invention
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Methods of treating fabry disease in pediatric patients. Provided are methods for the treatment o... |
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Invention
|
Neurotensin variants and tagged proteins comprising neurotensin or sortilin propeptide.
The disc... |
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Invention
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Neurotensin variants and tagged proteins comprising neurotensin or sortilin propeptide. The discl... |
|
|
Invention
|
Recombinant alpha-galactosidase a for treatment of fabry disease.
Described are compositions com... |
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Invention
|
Use of migalastat for treating fabry disease in pregnant patients.
Provided are methods of treat... |
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|
Invention
|
Augmented acid alpha-glucosidase for the treatment of pompe disease.
A method for treating Pompe... |
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|
Invention
|
Recombinant human acid alpha-glucosidase and uses thereof.
Provided herein are methods of treati... |
|
2021
|
P/S
|
Pharmaceutical preparations for the treatment of pompe
disease; pharmaceutical preparations for ... |
|
|
P/S
|
Pharmaceutical preparations for the treatment of Pompe
disease; Pharmaceutical preparations for ... |
|
|
Invention
|
Vesicle targeting proteins and uses of same.
The disclosure provides a novel method for treating... |
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Invention
|
Compositions useful for treatment of pompe disease.
Provided herein is a method for reducing the... |
|
2020
|
Invention
|
Recombinant cdkl5 proteins, gene therapy and production methods.
Compositions for CDKL5 gene the... |
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Invention
|
Method to predict response to pharmacological chaperone treatment of diseases.
The present inven... |
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|
Invention
|
Variant igf2 constructs.
Provided herein are novel IGF2 peptides, fusion proteins, and nucleic a... |
|
|
Invention
|
Method for capturing and purification of biologics.
Methods for the continuous production, captu... |
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|
P/S
|
Pharmaceutical preparations for the treatment of Pompe disease; pharmaceutical preparations for t... |
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|
P/S
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Providing health information in the field of rare genetic diseases; Providing medical information... |
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2019
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P/S
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Providing health information in the field of rare genetic diseases; providing medical information... |
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P/S
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Downloadable computer application software for mobile devices, namely, software for Fabry disease... |
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2018
|
P/S
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Activity kits comprised primarily of writing paper, adhesive
notepads, adhesive note paper, note... |
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2017
|
P/S
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Activity kits comprised primarily of writing paper, adhesive notepads, adhesive note paper, notep... |
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|
P/S
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Promoting public awareness of the need for treatment of rare genetic diseases, awareness of the i... |
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|
P/S
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Pharmaceutical preparations for the treatment of genetic
diseases. Performing diagnosis of disea... |
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|
P/S
|
Pharmaceutical preparations for the treatment of genetic diseases, namely lysosomal storage disor... |
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2016
|
P/S
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Downloadable software for use in the field of healthcare for lysosomal storage disorders, namely ... |
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P/S
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Charitable services, namely, providing financial assistance to patients with lysosomal storage di... |
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2015
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P/S
|
Pharmaceutical preparations; Pharmaceutical preparations for the treatment of Fabry disease. |
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|
P/S
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Pharmaceutical preparations for the treatment of Fabry disease |
|
|
P/S
|
Pharmaceutical preparations for the treatment of Fabry disease |
|
2014
|
P/S
|
Pharmaceutical preparations for the treatment of fabry
disease. |
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2006
|
P/S
|
Pharmaceutical preparations for the treatment of genetic diseases Performing diagnosis of disease... |
|
2005
|
P/S
|
Pharmaceutical preparations for the treatment of genetic diseases. Medical research, scientific r... |
