Abstract: This disclosure is directed to methods of methods of computing a composite measurement comprising employing two or more sub-instruments to measure one or more clinical symptoms of mitochondrial dysfunction or mitochondrial disease. This disclosure is also directed to methods of assessing and managing a subject with mitochondrial dysfunction or mitochondrial disease using a composite measurement.
Type:
Application
Filed:
January 19, 2024
Publication date:
July 11, 2024
Applicants:
RIBONOVA, INC., The Children's Hospital of Philadelphia
Inventors:
Marni J. Falk, Nigel L. Webb, Shana E. McCormack, Eric C. Yuen, Zarazuela Zolkipli-Cunningham, Anthony W. Ford-Hutchinson
Abstract: The present disclosure relates to apparatus and methods for safely and securely holding an infant and medical equipment proximal to the infant. In particular embodiments, an apparatus may comprise an infant support device configured to support an infant proximal to a user. Exemplary embodiments may also comprise a medical equipment support device configured to support medical equipment proximal to the infant.
Type:
Application
Filed:
March 9, 2021
Publication date:
June 27, 2024
Applicant:
The Children's Hospital of Philadelphia
Abstract: Provided herein are compositions and methods for delivering a molecular therapeutic to the cochlea of a subject. The methods comprise administering an adeno-associated virus (AAV) to the cerebrospinal fluid of the subject. The AAVs encode a therapeutic transgene for molecular therapy. Optionally, the therapeutic transgene may be operably linked to a cochlea-specific promoter.
Type:
Application
Filed:
April 27, 2022
Publication date:
June 20, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: Compositions and methods for treatment of mitochondrial respiratory chain dysfunction and other mitochondrial disorders are provided. Also disclosed are a number of screening assays having utility for the identification of agents which modulate the phenotype associated with mitochondrial respiratory chain dysfunction.
Abstract: This application relates to methods of diagnosing and treating anorexia nervosa (AN) and binge eating disorder (BED) with a nonselective activator of metabotropic glutamate receptors (mGluRs).
Type:
Application
Filed:
February 20, 2024
Publication date:
June 13, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: The present disclosure provides improved airway intervention devices that are coated with a material and contain an antimicrobial peptide. The devices are useful in controlling the microbial flora of the airway and preventing infections and conditions such as subglottic stenosis, pneumonia (VAP), laryngeal infection, post-operative dressing/packing-induced infection, upper airway infection, rhinosinusitis, choanal atresia, vocal fold injury and paralysis.
Type:
Application
Filed:
April 8, 2022
Publication date:
June 13, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors:
Riccardo L. GOTTARDI, Matthew R. ARONSON
Abstract: Compositions and methods for treatment of mitochondrial respiratory chain dysfunction and other mitochondrial disorders are provided. Also disclosed are a number of screening assays having utility for the identification of agents which modulate the phenotype associated with mitochondrial respiratory chain dysfunction.
Type:
Application
Filed:
November 17, 2023
Publication date:
June 6, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: An enema assist device is provided. The device includes a support and the support is configured to be placed on a toilet bowl such that the support front is oriented towards the front of the toilet bowl. The support is configured to support a fluid delivery tip such that the fluid delivery tip is disposed on the top surface of the support and protrudes at least partially beyond a plane defined by a top of the toilet seat.
Type:
Application
Filed:
March 22, 2022
Publication date:
May 16, 2024
Applicant:
The Children's Hospital of Philadelphia
Inventors:
Joy Kerr, Michelle Hsia, Jason Van Batavia
Abstract: This disclosure relates to the identification of a subset of mGluR network gene CNVs that are predictive of efficacy of treatment with fasoracetam, as well as the identification of an mGluR network gene CNV that is predictive of an increased likelihood of having ADHD as well as having certain symptoms associated with ADHD.
Type:
Application
Filed:
June 7, 2023
Publication date:
May 16, 2024
Applicant:
The Children's Hospital of Philadelphia
Inventors:
Garry A. Neil, Liza Squires, Hakon Hakonarson
Abstract: The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.
Type:
Application
Filed:
December 19, 2023
Publication date:
April 25, 2024
Applicant:
The Children's Hospital of Philadelphia
Abstract: Methods and uses for diagnosing and treating anxiety disorders are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Type:
Application
Filed:
October 30, 2023
Publication date:
March 28, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: The present disclosure is directed to antibodies binding to Glypican 2 and methods of using such antibodies to treat cancers that express or overexpress the Glypican 2 antigen.
Type:
Application
Filed:
October 31, 2023
Publication date:
March 14, 2024
Applicants:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA, THE GOVERNMENT OF THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY
Inventors:
John M. MARIS, Kristopher R. BOSSE, Dimiter DIMITROV, Zhongyu ZHU, Dontcho V. JELEV
Abstract: This invention relates to methods of treating and ameliorating congenital and neonatal hyperinsulinism and post-prandial hypoglycemia, comprising the step of administering an antagonist of the Glucagon-Like Peptide-1 (GLP-1) receptor, e.g. a GLP-1 fragment or analogue thereof.
Type:
Application
Filed:
February 14, 2023
Publication date:
March 14, 2024
Applicants:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, The Children's Hospital of Philadelphia
Inventors:
Doris STOFFERS, Diva D. DE LEON, Charles STANLEY
Abstract: Methods for diagnosing and treating conduct disorder are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.
Type:
Application
Filed:
October 30, 2023
Publication date:
March 7, 2024
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Abstract: The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.
Abstract: Compositions for the identification of patients at risk for the development of latent autoimmune diabetes, and methods of use thereof, for the diagnosis and treatment of the same.
Type:
Grant
Filed:
May 25, 2018
Date of Patent:
February 6, 2024
Assignee:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors:
Struan Frederick Grant, Rajashree Mishra