公开(公告)号：US20250017885A1

公开(公告)日：2025-01-16

申请号：US18770447

申请日：2024-07-11

Applicant: University of Kansas

Inventor： Duaa Jabari ,  Richard J. Barohn ,  Mazen M. Dimachkie ,  Heather Wilkins

IPC: A61K31/192 ,  C12Q1/6876 ,  G01N33/52

Abstract: A method for treating inclusion body myositis (IBM) in a subject can include: providing a subject that has IBM; administering phenylbutyrate (PBA) to the subject in a therapeutically effective amount to provide a treatment for the IBM; obtaining at least one biological sample from the subject after initiation of the treatment for the IBM; measuring a biological indicator in the at least one biological sample, wherein the biological indicator is mitochondrial membrane potential; and monitoring a change in the biological indicator as an indication of effectiveness of the treatment.

公开(公告)号：US20250025511A1

公开(公告)日：2025-01-23

申请号：US18779564

申请日：2024-07-22

Applicant: University of Kansas

Inventor： Richard J. Barohn ,  Mazen M. Dimachkie ,  Rupal Soder

IPC: A61K35/28 ,  C12N5/0775 ,  G01N33/68

Abstract: A method for treating inclusion body myositis (IBM) in a subject can include: providing a subject that has IBM; obtaining adipose-derived regenerative cells (ADRC); and administering the ADRC to the subject to treat the IBM. The ADRCs can be obtained by a closed system for use as the therapeutic as described herein. The ADRCs can be obtained in a system that can then be used for providing the ADRCs for administration. The system can be automated and may provide the ADRCs in sufficient purity for reimplantation. The method can include: injecting the ADRC as cells into one injection location or a plurality of injection locations to provide at least a million cells, more preferably at least 10 million cells, even more preferably at least 20 million cells, or most preferably at least 30 million cells.