首页> Stem cell reviews and reports> Long-Term Safety and Efficacy of Systemic DT-DEC01 Cell Therapy in Non-Ambulatory Duchenne Muscular Dystrophy Patients: a 24-Month Clinical Evaluation.

Long-Term Safety and Efficacy of Systemic DT-DEC01 Cell Therapy in Non-Ambulatory Duchenne Muscular Dystrophy Patients: a 24-Month Clinical Evaluation.

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第一作者： Maria,Siemionow

第一单位： Chair and Department of Traumatology, Orthopaedics and Surgery of the Hand, Poznan University of Medical Sciences, Poznan, 61‑545, Poland. siemiom@dystrogen.com.;Dystrogen Therapeutics Corp, Chicago, IL, 60609, USA. siemiom@dystrogen.com.;Department of Orthopaedics, University of Illinois at Chicago, Chicago, IL, 60612, USA. siemiom@dystrogen.com.

作者： Maria,Siemionow ^[1] ; Grzegorz,Biegański ^[2] ; Adam,Niezgoda ^[3] ; Agnieszka,Sobkowiak-Sobierajska ^[4] ; Jarosław,Czarnota ^[5] ; Krzysztof,Siemionow ^[6] ; Anna,Ziemiecka ^[7] ; Katarzyna,Bożyk ^[7] ; Jacek,Wachowiak ^[4]

作者单位： Chair and Department of Traumatology, Orthopaedics and Surgery of the Hand, Poznan University of Medical Sciences, Poznan, 61‑545, Poland. siemiom@dystrogen.com.;Dystrogen Therapeutics Corp, Chicago, IL, 60609, USA. siemiom@dystrogen.com.;Department of Orthopaedics, University of Illinois at Chicago, Chicago, IL, 60612, USA. siemiom@dystrogen.com. ^[1] Department of Infectious Diseases and Child Neurology, Poznan University of Medical Sciences, Poznan, 60‑572, Poland. ^[2] Department of Neurology, Poznan University of Medical Sciences, Poznan, 60‑355, Poland. ^[3] Department of Pediatric Oncology, Hematology and Transplantology, Poznan University of Medical Sciences, Poznan, 60‑572, Poland. ^[4] Hospital MedPolonia, Poznan, 60‑693, Poland. ^[5] Dystrogen Therapeutics Corp, Chicago, IL, 60609, USA.;Department of Orthopaedics, University of Illinois at Chicago, Chicago, IL, 60612, USA. ^[6] Dystrogen Therapeutics Corp, Chicago, IL, 60609, USA. ^[7]

关键词 DT-DEC01 therapy Duchenne muscular dystrophy (DMD)Dystrophin Expressing Chimeric (DEC) cells Long-term efficacy Safety Stem cell therapy

医学主题词人类(Humans);肌营养不良, 杜氏(Muscular Dystrophy, Duchenne);青少年(Adolescent);儿童(Child);男(雄)性(Male);肌营养不良蛋白(Dystrophin);治疗结果(Treatment Outcome)

DOI 10.1007/s12015-025-11039-8

PMID 41410800

发布时间 2026-01-30

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