Duchenne muscular dystrophy: current treatments and tissue engineering strategies

Duchenne muscular dystrophy (DMD)is an X-linked recessive disorder characterized by progressive muscle degeneration and weakness. Nearly all cases of DMD occur in male children. It remains a challenging condition with no cure, requiring ongoing research and advanced medical care to manage symptoms and improve quality of life. However, the increasing market demand and investment in DMD therapeutics are driven by a more favorable regulatory environment, advances in gene-editing technologies, and significant progress in clinical trial drug developments. Recent clinical trials using cardiosphere-derived cells (CDCs) have shown promise in preventing heart and muscle weakening in non-ambulatory patients, indicating a potential breakthrough in tissue engineering solutions for management of DMD. Furthermore, innovations in tissue engineering, including the use of stem cells and gene therapy are paving the way for novel therapeutic strategies designed for muscle regeneration and functional restoration. This review paper explores healthy muscle physiology, the pathophysiology of DMD and emerging market trends. Furthermore, this paper discusses past and ongoing clinical trials regarding tissue engineering solutions for DMD.

Authors

Publication Details

Journal
Journal of Applied Biotechnology & Bioengineering
Published
2024-11-06
DOI
https://doi.org/10.15406/jabb.2024.11.00375
Primary Topic
Muscle Physiology and Disorders
Type
article
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article

Duchenne muscular dystrophy: current treatments and tissue engineering strategies

Charmaine Tan, Bill Tawil
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Journal of Applied Biotechnology & Bioengineering
Muscle Physiology and Disorders
article

Duchenne muscular dystrophy: current treatments and tissue engineering strategies

Charmaine Tan, Bill Tawil
article en

Abstract

Duchenne muscular dystrophy (DMD)is an X-linked recessive disorder characterized by progressive muscle degeneration and weakness. Nearly all cases of DMD occur in male children. It remains a challenging condition with no cure, requiring ongoing research and advanced medical care to manage symptoms and improve quality of life. However, the increasing market demand and investment in DMD therapeutics are driven by a more favorable regulatory environment, advances in gene-editing technologies, and significant progress in clinical trial drug developments. Recent clinical trials using cardiosphere-derived cells (CDCs) have shown promise in preventing heart and muscle weakening in non-ambulatory patients, indicating a potential breakthrough in tissue engineering solutions for management of DMD. Furthermore, innovations in tissue engineering, including the use of stem cells and gene therapy are paving the way for novel therapeutic strategies designed for muscle regeneration and functional restoration. This review paper explores healthy muscle physiology, the pathophysiology of DMD and emerging market trends. Furthermore, this paper discusses past and ongoing clinical trials regarding tissue engineering solutions for DMD.

Journal of Applied Biotechnology & BioengineeringVol. 11(6)
Openalex Percentile: Top 81%
Muscle Physiology and Disorders
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Duchenne muscular dystrophy: current treatments and tissue engineering strategies — Charmaine Tan, Bill Tawil · Journal of Applied Biotechnology & Bioengineering (2024) | TGRS Research Map | TGRS