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:
October 28, 2022
Publication date:
May 11, 2023
Applicant:
The Children's Hospital of Philadelphia
Abstract: Provided are macromolecular prodrugs in which camptothecin analogs are covalently bonded to polymers via ester bonds that are labile under physiological conditions. Also provided are methods of treating cancer, especially neuroblastoma with the macromolecular prodrugs.
Type:
Grant
Filed:
February 11, 2022
Date of Patent:
May 9, 2023
Assignee:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors:
Michael Chorny, Ivan Alferiev, Garrett M. Brodeur
Abstract: A co-drug according to Formula (I) or Formula (II) is provided, R—X—NH—CO—CO—OR1??(I) R—X—CO—O—CH2—CO—OR1??(II) wherein R is a tocol moiety, a tocol analog moiety, or a capsaicinoid moiety; X is a direct bond or a linking group; and OR1 is the residue of an anticancer or antirestenotic agent bearing at least one hydroxyl group by which the CO—OR1 ester linkage is formed. Nanoparticles that include the abovementioned co-drug are also provided, as well as a method of treating a cancer patient that includes administering an effective amount of the co-drug or nanoparticles.
Type:
Grant
Filed:
March 13, 2018
Date of Patent:
April 25, 2023
Assignee:
The Children's Hospital of Philadelphia
Inventors:
Ivan Alferiev, Michael Chorny, Garrett M. Brodeur
Abstract: The disclosure is directed to crystalline forms and mixtures thereof of R-fasoracetam. Such crystalline forms include Form II monohydrate R-fasoracetam and the anhydrate form of R-fasoracetam. The disclosure further includes mixtures of the anhydrate form of R-fasoracetam together with one or more of Form I monohydrate R-fasoracetam and Form II monohydrate R-fasoracetam.
Type:
Application
Filed:
November 30, 2022
Publication date:
March 23, 2023
Applicant:
The Children's Hospital of Philadelphia
Abstract: The disclosure is directed to cocrystals of fasoracetam, including R-fasoracetam, and various coformers. Crystalline materials comprising fasoracetam, including R-fasoracetam, are also provided. The disclosure further includes pharmaceutical compositions and methods of treatment of the cocrystals and crystalline materials of the disclosure.
Type:
Grant
Filed:
January 17, 2019
Date of Patent:
March 21, 2023
Assignee:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors:
Tom Leyssens, Richard Alan Couch, Michael Paul Rene Guillot, Bram Harmsen, Thomas R. Bailey, Martin Appelmans
Abstract: Algorithms for detecting endotracheal intubation and/or misplacement of endotracheal tubes in child patients during anesthesia for use with anesthesia machines, mechanical ventilators, and/or respiratory function monitors. An algorithm uses end-tidal carbon dioxide (EtCO2), and tidal volume (TV) or peak inspiratory pressure (PIP) to detect exact intubation time. Another algorithm uses respiratory parameters to identify and/or confirm the type of airway device used during mechanical ventilation, and to detect if and when an issue has arisen with use of a specific airway device to provide real-time decision support to attending medical care professionals.
Abstract: The invention relates to compounds of Formula I or Formula II: to compositions comprising such compounds, and to methods of use thereof for treating neurodegenerative disorders, such as Friedreich ataxia.
Type:
Application
Filed:
November 12, 2020
Publication date:
January 26, 2023
Applicants:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, The Children's Hospital of Philadelphia
Inventors:
Amos SMITH, Robert B. WILSON, Donna M Huryn, Maria Grazia Cotticelli, Shujuan XIA, Taehee LEE, Roberto FORESTIERI
Abstract: The disclosure is directed to crystalline forms and mixtures thereof of R-fasoracetam. Such crystalline forms include Form II monohydrate R-fasoracetam and the anhydrate form of R-fasoracetam. The disclosure further includes mixtures of the anhydrate form of R-fasoracetam together with one or more of Form I monohydrate R-fasoracetam and Form II monohydrate R-fasoracetam.
Abstract: A power tool, such as a cast saw is disclosed. The cast saw is equipped with at least one thermocouple and and/or at least one infrared detector that measure the temperature of the cutting blade on the cast saw or the area in the cast immediately surrounding the cutting blade. The cast saw is also equipped with at least one LED that can alert a user of the cast saw when blade or the area in the cast immediately surrounding the blade is approaching or has reached a temperature that may burn a patient. Also disclosed is a shroud for the cutting blade of the cast saw. The shroud not only surrounds at least part of the blade but also houses the thermocouple or infrared detector and the LEDs(s). A method of operating the cast saw to cut a cast off of a patient is also disclosed.
Type:
Application
Filed:
September 25, 2020
Publication date:
December 22, 2022
Applicant:
The Children's Hospital of Philadelphia
Inventors:
Bernard David HORN, John Todd Rutter LAWRENCE
Abstract: Devices and methods for management of a guidewire during catheter insertion. Particular embodiments include unidirectional valves to allow movement of a guidewire in one direction only.
Type:
Application
Filed:
July 9, 2020
Publication date:
November 17, 2022
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 and methods for the diagnosis and treatment of POTS are disclosed. In one aspect, a method for identifying a human subject having a predisposition for, or having, postural orthostatic tachycardia syndrome (POTS) is provided. An exemplary embodiment comprises obtaining a nucleic acid sample from said subject; detecting a nucleic acid having one or more single nucleotide polymorphisms (SNPs) in the locus encoding PPP1R12B selected from rs12741415 and/or GSA-rs116062217, or a SNP in linkage disequilibrium with one or more of the SNPs, by contacting the nucleic acid sample with a probe or primer of sufficient length and composition to detect the SNP; and identifying the subject as having a predisposition for POTS if one or more SNPs are identified.
Type:
Application
Filed:
September 9, 2020
Publication date:
November 3, 2022
Applicant:
THE CHILDREN'S HOSPITAL OF PHILADELPHIA