An analysis revealed global trends and emerging focus areas in Duchenne muscular dystrophy (DMD) research, emphasizing the growing impact of precision medicine and gene therapies.

Heart anatomy on paper | Image credit: larisikstefania – stock.adobe.com

MRI Biomarker May Signal Early Heart Damage in DMD

By Hayden E. Klein

December 20th 2024

Septal circumferential strain (Ecc) was significantly decreased in LGE-negative boys with Duchenne muscular dystrophy (DMD) and correlated to ventricle changes.

Gene therapy | Image credit: Treecha – stock.adobe.com

FDA Pauses PepGen’s DMD Drug Trial in the US

By Hayden E. Klein

December 19th 2024

The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.

Top 5 Duchenne Muscular Dystrophy Content of 2024

By Hayden E. Klein

December 19th 2024

From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and research.

Gene therapy graphic with DNA strands | Image credit: natali_mis – stock.adobe.com

Real-World Challenges in DMD PMO Treatment

By Hayden E. Klein

November 25th 2024

A claims-based study revealed high adherence to phosphorodiamidate morpholino oligomer (PMO) treatments for Duchenne muscular dystrophy (DMD), but highlights limitations in interpreting real-world effectiveness from claims data.

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