Great strides have been made in Duchenne muscular dystrophy treatment in recent years. In Russia, two of the three new drug types are now available, offering fresh options for patients. A leading pediatric neurologist in the Moscow region discussed these developments in an interview, highlighting the evolving landscape of care for Duchenne.
Experts anticipate that the first gene replacement therapy could be licensed and ready for use by mid-2023, representing a major shift in how the disease is managed. This approach involves a one-time intervention that targets the defective gene and initiates a corrective process. If successful, it may prevent disease progression or slow it significantly, reducing genetic damage compared with traditional therapies.
Historically, progressive Duchenne muscular dystrophy was managed primarily with hormonal therapies, which offered limited benefit and extended life by roughly two years on average. As new treatment modalities emerge, clinicians see the potential to improve outcomes for a larger portion of patients, with estimates suggesting gene replacement therapy could be suitable for about half of those affected.
Access to these advanced therapies is a topic of active discussion, with patient support organizations and foundations playing a role in expanding availability. National health programs and charitable foundations are involved in addressing barriers to treatment and ensuring broader patient access where possible.
Progressive Duchenne muscular dystrophy is a serious inherited condition that predominantly affects boys. Symptoms typically appear in childhood, with loss of ambulation occurring around ages eight to ten and the emergence of significant complications by adolescence. Ongoing research aims to prolong independent living and reduce the burden of disease through innovative therapies and comprehensive care.
In the past, scientists reported progress in microgene technologies aimed at treating muscular dystrophy. While these efforts hold promise, the field continues to evolve, with researchers pursuing strategies to refine gene delivery, safety, and long-term effectiveness. Parallel lines of study also explore cardiac and other organ involvement, seeking holistic improvements in patient health and quality of life.
Earlier work by Russian researchers has shown potential in restoring cardiac cells in other conditions, illustrating the broader impact of gene-based and cell-based approaches. The ongoing exploration of such techniques demonstrates a growing commitment to translating laboratory findings into real-world therapies for Duchenne and related disorders.